Pyruvate Kinase Deficiency

– U.S. Regulatory Filing Under Active Review for Approval of PYRUKYND® (mitapivat) in Thalassemia, with PDUFA Goal Date of September 7, 2025 – – Phase 3 RISE UP Study of Mitapivat in Sickle Cell Disease On Track, with Topline Results Expected in Late 2025; Potential U.S. Commercial Launch in 2026 – – Tebapivat Advancing in Clinical Trials for Lower-Risk Myelodysplastic Syndromes (LR-MDS) and Sickle Cell Disease – – PYRUKYND Net Revenue of $8.7 Million in Q1; Cash, Cash Equivalents and Marketable Securities of $1.4 Billion as of March 31, 2025 – CAMBRIDGE, Mass. , May 01, 2025 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today reported business highlights and financial results for the first quarter ended March 31, 2025 . “We are pleased with our strong start to 2025, highlighted by the acceptance of our sNDA for thalassemia with a PDUFA goal date of September 7, 2025 . Our engagement with the FDA is progressing as expected, and we are committed to bringing PYRUKYND to thalassemia patients, irrespective of genotype or transfusion needs,” said Brian Goff , chief executive officer at Agios. “Looking ahead, our focus is also on delivering the topline results from the Phase 3 RISE UP study in sickle cell disease, which remains on track for year-end, and continuing to advance our early and mid-stage clinical programs. Supported by our strong financial position and highly experienced team, we are driving forward PYRUKYND’s multi-billion-dollar potential while building a pipeline designed for lasting impact, with the goal of creating significant value for shareholders and delivering transformative therapies for patients.” First Quarter 2025 and Recent Highlights PYRUKYND® Revenues: Generated $8.7 million in net revenue for the first quarter of 2025, compared to $8.2 million in the first quarter of 2024. A total of 234 unique patients have completed prescription enrollment forms, representing an increase of 5 percent over the fourth quarter of 2024. A total of 136 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy, as compared to 130 patients at the end of the fourth quarter 2024. Thalassemia: The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing. Sickle Cell Disease: The Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older continued to progress as anticipated. This 52-week Phase 3 study completed enrollment in October 2024 , enrolling more than 200 patients worldwide. Advanced final preparations to initiate a Phase 2 clinical trial of tebapivat in patients with sickle cell disease in mid-2025. Pediatric Pyruvate Kinase (PK) Deficiency: Reported positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Safety was consistent with the profile for mitapivat previously observed in adults with PK deficiency who are not regularly transfused. ACTIVATE-Kids is the first study to demonstrate efficacy of an oral therapy for children with PK Deficiency who are not regularly transfused. Lower-risk Myelodysplastic Syndromes (LR-MDS): Progressed patient enrollment in the Phase 2b study of tebapivat in LR-MDS. Corporate: Krishnan Viswanadhan , Pharm. D, joined Agios as Chief Corporate Development and Strategy Officer, responsible for leading the company’s corporate strategy, business development, and long-term growth initiatives. Previously, he served as President and Chief Operating Officer of Be Biopharma and at various senior roles at both Bristol Myers Squibb and Celgene. The U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025. The FDA has communicated that at this time no advisory committee meeting is planned, and the review is ongoing. The Phase 3 RISE UP study evaluating mitapivat for the treatment of sickle cell disease patients who are 16 years of age or older continued to progress as anticipated. This 52-week Phase 3 study completed enrollment in October 2024 , enrolling more than 200 patients worldwide. Advanced final preparations to initiate a Phase 2 clinical trial of tebapivat in patients with sickle cell disease in mid-2025. Reported positive topline results from the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused. Safety was consistent with the profile for mitapivat previously observed in adults with PK deficiency who are not regularly transfused. ACTIVATE-Kids is the first study to demonstrate efficacy of an oral therapy for children with PK Deficiency who are not regularly transfused. Progressed patient enrollment in the Phase 2b study of tebapivat in LR-MDS. Key Upcoming Milestones & Priorities Agios expects to achieve the following key milestones in 2025: Thalassemia: Receive FDA regulatory decision for PYRUKYND for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia (PDUFA goal date is September 7, 2025). Continue progressing the review of regulatory applications with health authorities in the European Union , Kingdom of Saudi Arabia and United Arab Emirates . Sickle Cell Disease: Announce topline results from the Phase 3 RISE UP study of mitapivat in sickle cell disease in late 2025, with a potential U.S. commercial launch in 2026. Additionally, begin patient enrollment for the Phase 2 study of tebapivat in sickle cell disease in mid-2025. LR-MDS: Complete patient enrollment in the Phase 2b study of tebapivat for LR-MDS in late 2025. Early-Stage Pipeline: File an Investigational New Drug Application for AG-236, an siRNA targeting TMPRSS6 intended for the treatment of polycythemia vera, in mid-2025. First Quarter 2025 Financial Results Revenue: Net product revenue from sales of PYRUKYND for the first quarter of 2025 was $8.7 million , compared to $8.2 million for the first quarter of 2024. Cost of Sales: Cost of sales for the first quarter of 2025 was $1.1 million . Research and Development (R&D) Expenses: R&D expenses were $72.7 million for the first quarter of 2025, compared to $68.6 million for the first quarter of 2024. The year-over-year increase was primarily attributed to an increase in workforce-related expenses and costs associated with clinical trials of tebapivat in LR-MDS and sickle cell disease, partially offset by lower costs associated with the clinical trials of mitapivat in thalassemia and pediatric PKD. Selling, General and Administrative (SG&A) Expenses: SG&A expenses were $41.5 million for the first quarter of 2025 compared to $31.0 million for the first quarter of 2024. The year-over-year increase was primarily attributable to an increase in commercial-related activities, including headcount, as the company prepares for the potential approval of PYRUKYND in thalassemia. Net Loss: Net loss was $89.3 million for the first quarter of 2025 compared to $81.5 million for the first quarter of 2024. Cash Position and Guidance: Cash, cash equivalents and marketable securities as of March 31, 2025 , were $1.4 billion compared to $1.5 billion as of December 31, 2024 . Agios expects that its cash, cash equivalents and marketable securities, together with anticipated product revenue and interest income, will provide the financial independence to prepare for potential PYRUKYND launches in thalassemia and sickle cell disease, advance existing programs, and to opportunistically expand its pipeline through both internally and externally discovered assets. Conference Call Information Agios will host a conference call and live webcast today at 8:00 a.m. ET to discuss the company’s first quarter 2025 financial results and recent business highlights. The live webcast will be accessible on the Investors section of the company's website (www.agios.com) under the “Events & Presentations” tab. A replay of the webcast will be available on the company’s website approximately two hours after the event. About Agios Agios is the pioneering leader in PK activation and is dedicated to developing and delivering transformative therapies for patients living with rare diseases. In the U.S. , Agios markets a first-in-class pyruvate kinase (PK) activator for adults with PK deficiency, the first disease-modifying therapy for this rare, lifelong, debilitating hemolytic anemia. Building on the company's deep scientific expertise in classical hematology and leadership in the field of cellular metabolism and rare hematologic diseases, Agios is advancing a robust clinical pipeline of investigational medicines with programs in alpha- and beta-thalassemia, sickle cell disease, pediatric PK deficiency, myelodysplastic syndromes (MDS)-associated anemia and phenylketonuria (PKU). In addition to its clinical pipeline, Agios is advancing a preclinical TMPRSS6 siRNA as a potential treatment for polycythemia vera. For more information, please visit the company’s website at www.agios.com. Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the potential benefits of PYRUKYND® (mitapivat), tebapivat, AG-236 and AG-181; Agios’ plans, strategies and expectations for its preclinical, clinical and commercial advancement of its drug development, including PYRUKYND®, tebapivat, AG-236 and AG-181; Agios’ use of proceeds from the transaction with Royalty Pharma; potential U.S. net sales of vorasidenib and potential future royalty payments; Agios’ strategic vision and goals, including its key milestones for 2025; and the potential benefits of Agios’ strategic plans and focus. The words “anticipate,” “expect,” “goal,” “hope,” “milestone,” “plan,” “potential,” “possible,” “strategy,” “will,” “vision,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from Agios’ current expectations and beliefs. For example, there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases, or that development of any of Agios’ product candidates will successfully continue. There can be no guarantee that any positive developments in Agios’ business will result in stock price appreciation. Management's expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other important factors, including, without limitation: risks and uncertainties related to the impact of pandemics or other public health emergencies to Agios’ business, operations, strategy, goals and anticipated milestones, including its ongoing and planned research activities, ability to conduct ongoing and planned clinical trials, clinical supply of current or future drug candidates, commercial supply of current or future approved products, and launching, marketing and selling current or future approved products; Agios’ results of clinical trials and preclinical studies, including subsequent analysis of existing data and new data received from ongoing and future studies; the content and timing of decisions made by the U.S. FDA, the EMA or other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies; Agios’ ability to obtain and maintain requisite regulatory approvals and to enroll patients in its planned clinical trials; unplanned cash requirements and expenditures; competitive factors; Agios' ability to obtain, maintain and enforce patent and other intellectual property protection for any product candidates it is developing; Agios’ ability to establish and maintain key collaborations; uncertainty regarding any royalty payments related to the sale of its oncology business or any milestone or royalty payments related to its in-licensing of AG-236, and the uncertainty of the timing of any such payments; uncertainty of the results and effectiveness of the use of Agios’ cash and cash equivalents; and general economic and market conditions. These and other risks are described in greater detail under the caption "Risk Factors" included in Agios’ public filings with the Securities and Exchange Commission . Any forward-looking statements contained in this press release speak only as of the date hereof, and Agios expressly disclaims any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Contacts: Investor Contact Chris Taylor , VP, Investor Relations and Corporate Communications Agios Pharmaceuticals IR@agios.com Media Contact Eamonn Nolan , Senior Director, Corporate Communications Agios Pharmaceuticals media@agios.com Source: Agios Pharmaceuticals, Inc.

Check back to this tracker for regulatory updates on marketed drugs, including expansions into new geographies and indications. Welcome to Fierce Pharma's regulatory tracker for the first half of 2025. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Be sure to come back regularly for the latest updates.UPDATED: Friday, March 14 at 9:25 a.m. ETChina's National Medical Products Administration (NMPA) has approved Innovent's Sycume as a treatment for thyroid eye disease (TED).The drug, also known as teprotumumab N01, becomes the first anti-insulin-like growth factor 1 receptor (IGF-1R) antibody to score approval in the country.In a press release, Innovent said the drug's approval "has ended a 70-year drought of no new treatment option for TED in China."In the U.S., teprotumumab is marketed by Amgen as Tepezza. In Europe, officials have expanded the label for Bristol Myers Squibb's cell therapy Breyanzi.Specifically, the European Commission approved the drug for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.The approval is based on results from the global phase 2 study called TRANSCEND FL, which enrolled patients with relapsed or refractory indolent non-Hodgkin lymphoma (NHL), including FL. In the third-line-plus treatment setting, 97.1% of patients responded to the drug, and 94.2% of patients achieved a complete response.Before this approval, Breyanzi had scored EU nods to treat certain patients with diffuse large B-cell lymphoma, high grade B-cell lymphoma and primary mediastinal large B-cell lymphoma.UPDATED: Thursday, March 13 at 8:30 a.m. ETKelun-Biotech's TROP 2 ADC sacituzumab tirumotecan (sac-TMT) has scored its second approval in China.The country's National Medical Products Administration (NMPA) approved the drug to treat certain adults with EGFR-positive locally advanced or metastatic non-squamous non-small cell lung cancer (NSCLC). The nod covers the drug's use in patients who have progressed after treatment with EGFR-tyrosine kinase inhibitor (TKI) therapy and platinum-based chemotherapy, Kelun said in a press release.With the approval, sac-TMT becomes the first TROP2 ADC approved to treat lung cancer anywhere in the world, according to the company.In a pivotal study, the drug "demonstrated a statistically significant and clinically meaningful improvement" in objective response rate, progression-free survival and overall survival compared with docetaxel, Kelun said. The study tested the drugs in patients with locally advanced or metastatic EGFR-mutant NSCLC who failed after prior treatment with an EGFR-TKI and platinum-based chemo.The approval is sac-TMT's second in the country after the NMPA approved it to treat certain patients with  triple negative breast cancer last year.In 2022, Merck & Co. licensed ex-China rights to the drug.UPDATED: Friday, March 7 at 9:35 a.m. ETThe European Commission (EC) has signed off on Bristol Myers Squibb’s immunotherapy combination of Opdivo and Yervoy to treat first-line patients with advanced or unresectable hepatocellular carcinoma (HCC).The nod was based on Opdivo and Yervoy showing an overall survival edge in the CheckMate-9DW trial over an investigator's choice of oral treatments.The Opdivo-Yervoy combo also faces an FDA decision date of April 21 to treat unresectable HCC. In 2020, the FDA granted accelerated approval to Opdivo-Yervoy to treat second-line advanced HCC.Opdivo and Yervoy were originally approved to treat melanoma, in 2014 and 2011 respectively. In combination, they have been blessed by the FDA in four other cancer types: renal cell carcinoma (2018), colorectal cancer (2018), mesothelioma (2020) and lung cancer (2020).Approximately 90% of liver cancer diagnoses are of the HCC type, BMS said. Liver cancer is the world’s third leading cause of cancer deaths.The FDA has expanded its approval for ALK’s immunotherapy Odactra for house dust mite (HDM) allergies to children ages 5 through 11. The under-the-tongue tablet was originally approved for adults in 2017 and then for adolescents ages 12 to 17 in 2023.UPDATED: Thursday, March 6 at 9:45 a.m. ETARS Pharmaceuticals needle-free epinephrine option, neffy nasal spray, can now be used to treat type 1 allergic reactions including anaphylaxis in children.To win the pediatric nod from the FDA, ARS ran studies proving that pharmacokinetic and pharmacodynamic responses from neffy in both children and adults were consistent with that of standard epinephrine injection products. The label expansion comes after the drug’s August approval as the first major innovation in epinephrine delivery in more than 35 years. Needle-free delivery is especially significant in the pediatric population as many children and caregivers fear needle-based auto-injectors, standing to delay lifesaving treatment.Human factor studies also show that children as young as 10 can use the nasal spray effectively and that untrained individuals, such as babysitters or teachers, can also correctly administer the drug. In the U.S., nearly 4 out of every 10 epinephrine prescriptions are for children under the age of 18 and some one in 13 children have severe food allergies, with more than 40% having experienced severe reactions, according to ARS. UPDATED: Wednesday, March 5 at 10:10 a.m. ETRoche's supplemental biologics application for Gazyva to treat lupus nephritis has been accepted for review by the FDA, the company said in a Wednesday press release.The filing leverages results from the phase 3 REGENCY study, which showed that 46.4% of patients with lupus nephritis who received Gazyva plus standard care achieved a complete renal response after 76 weeks. For patients who received standard care alone, the number was 33.1%, according to study results published last month.First approved in 2013, Gazyva is already cleared to treat chronic lymphocytic leukemia and follicular lymphoma in certain situations.The FDA is expected to make a decision on the lupus nephritis filing by October, according to Roche.UPDATED: Monday, March 3 at 4:30 p.m. ETDespite a reconsideration after a prior rejection, Australian drug regulators ultimately passed on Eisai's BioArtic-partnered Alzheimer’s disease treatment Leqembi. The Therapeutic Goods Administration (TGA) of Australia first decided against approval for patients with early Alzheimer’s disease in October. Eisai then requested reconsideration of the decision in December, proposing the same narrow indication given to the drug by the Medicines and Healthcare products Regulatory Agency (MHRA) and European Medicines Agency (EMA). Those approvals stipulate that Leqembi can only be used in patients with one or no copies of the ApoE4 gene due to safety concerns linking two copies of the gene to increased risks of brain swelling and bleeding, a side effect known as Amyloid Related Imaging Abnormalities (ARIAs).However, the TGA did not agree with the safety profile of Leqembi in ApoE4 heterozygotes, or those with one copy of the ApoE4 gene. Instead, the regulator offered an alternative narrow indication covering only ApoE4 noncarriers. Eisai floated other indications instead, including one that would keep the ApoE4 noncarrier and heterozygote patient populations but require heterozygotes to receive treatment in specialist centers with expert physician supervision. Still, the TGA rejected those options.BioArtic, Eisai’s long-time research partner that originally developed the antibody that became Leqembi, said it’s “very disappointed” by TGA’s decision, CEO Gunilla Osswald said in a statement. Eisai said it remains committed to ensuring eligible Australians can access the treatment and is “exploring options” to achieve this including a potential review by Australia’s Administrative Review Tribunal. UPDATED: Friday, Feb. 28 at 10:30 a.m. ETThe CDC said it's investigating five hospitalizations related to heart and nervous system events following vaccination with Valneva's Ixchiq among people 65 years and older. Ixchiq became the first FDA-approved chikungunya vaccine in November 2023. The CDC currently recommends the live-attenuated shot to be used for adults traveling to a country or territory where there is a chikungunya outbreak. Based on information from the CDC, at least four of the five individuals had also received other vaccines, and most of them had significant underlying medical conditions, a Valneva spokesperson said in a statement to Fierce Pharma."Valneva has not identified any safety signal concerns through post-marketing safety monitoring, including periodic safety reports and routine signal detection activities, and Ixchiq’s safety profile remains consistent with clinical trial data, unchanged and positive," the spokesperson said.The CDC said findings from its investigation will be discussed with experts at an Advisory Committee on Immunization Practices (ACIP) meeting. This year's first ACIP meeting, which was supposed to take place this week, was postponed with no new time provided by the CDC.The U.K. has approved Moderna's respiratory syncytial virus (RSV) vaccine mRESVIA to protect patients ages 60 and over against lower respiratory tract disease caused by the virus.In a study conducted in about 37,000 older adults, people who received mRESVIA had a 79% reduction in their risk of getting lower respiratory tract disease caused by RSV, compared with those who received placebo, around four months after vaccination.The shot will be manufactured at the Moderna Innovation and Technology Centre in Oxfordshire, which will be fully operational later this year, CEO Stéphane Bancel said in a statement.As is the case in the U.S., Moderna's mRNA shot will compete with GSK's Arexvy and Pfizer's Abrysvo in the U.K. as well.UPDATED: Thursday, Feb. 27 at 8:32 a.m. ETAfter being turned away by the FDA nearly one year ago, Regeneron this week said that the U.S. agency has accepted for review its biologics license application for bispecific antibody odronextamab.The FDA is expected to act on the application by July 30, Regeneron said in a Wednesday press release.In March of 2024, the agency rejected an accelerated approval filing for the drug based on the timeline of Regeneron's ongoing confirmatory trials. That decision spurred some confusion at Regeneron—and beyond—about a new requirement from the agency that confirmatory trials be "well underway" by the time it awards accelerated approvals.Early this year, the FDA sought to clear up the issue with new guidance on its expectations around accelerated approvals and confirmatory trial timelines.Nevertheless, Regeneron's review for the drug to treat patients with relapsed/refractory (R/R) follicular lymphoma (FL) after two or more lines of systemic therapy now appears back on track. In its latest press release, Regeneron said the resubmission "follows the achievement of an FDA-mandated enrollment target for the Phase 3 confirmatory trial." As the company notes, the enrollment target was the "sole approvability issue identified by the FDA" in its last submission.In Europe, regulators last year approved the drug for patients with R/R FL and R/R diffuse large B-cell lymphoma after two or more lines of systemic therapy. The medicine carries the brand name Ordspono in the region.In a phase 2 study, 80% of patients with FL who were treated with odronextamab responded to the therapy, with 74% achieving a complete response.UPDATED: Tuesday, Feb. 25 at 9:30 a.m. ETMerck's Keytruda, already boasting some 40 FDA approvals, has picked up a priority review in a proposed new use.The company's filing for the drug as part of a perioperative regimen for patients with resectable locally advanced head and neck squamous cell carcinoma has been accepted for a speedy review, Merck said on Tuesday.The filing seeks approval for Keytruda as a neoadjuvant treatment prior to surgery, followed by its administration as an adjuvant treatment in combination with standard of care radiotherapy—with or without cisplatin—after surgery. Afterward, the company's filing proposes Keytruda being used as a single agent.Merck's application leverages results from the KEYNOTE-689 trial, which showed that the perioperative regimen significantly reduced the risk of disease recurrence, worsening or death in certain patients with resected HNSCC compared with traditional postsurgical radiotherapy alone.The FDA is expected to act on the application by June 23.In Japan, Sanofi's Sarclisa has picked up an approval to treat newly diagnosed patients with multiple myeloma.Japan's Ministry of Health, Labour and Welfare (MHLW) approved the drug as part of a combination with bortezomib, lenalidomide, and dexamethasone (VRd) in the patient group based on results from the phase 3 IMROZ trial. The study showed that Sarclisa's addition to VRd significantly improved progression-free survival compared with VRd alone in patients with newly diagnosed, transplant-ineligible multiple myeloma.Sarclisa launched in 2020 in Japan and is approved as a part of four different regimens in the country, Sanofi noted in a press release.UPDATED: Monday, Feb. 24 at 10:52 a.m. ETGilead's application for twice-yearly lenacapavir has been accepted by the European Medicines Agency for accelerated review as an HIV pre-exposure prophylaxis (PrEP) option. Simultaneously, the drug will be assessed under EU-Medicines for all, which can be used to facilitate expedited review processes in countries outside the EU, including in low- and lower-middle-income countries. The FDA has put lenacapavir's PrEP application under priority review last week with a target decision date set for June 19, 2025.Several companies have launched their biosimilar products referencing Johnson & Johnson's star inflammatory disease drug Stelara in the U.S., following Amgen's January launch of Wezlana as the first biosimilar referencing the star IL-12/23 inhibitor. Teva and Alvotech just rolled out Selarsdi covering all indications and presentations of the original Stelara. Teva can start to claim interchangeability after an exclusivity period for Wezlana ends on April 30. Sandoz announced the launch of its Samsung Bioepis-partnered Pyzchiva, which the Swiss company views as a key driver in its global growth strategy. Sandoz touts Pyzchiva's extended stability, including the ability to be re-refrigerate, as an advantage over the original Stelara.In addition, Biocon Biologics has also entered the U.S. Stelara biosimilar market with Yesintek. It marks the Biocon subsidiary's "first product launch in the United States since becoming a fully integrated global biosimilars organization," CEO Shreehas Tambe said in a release. The FDA has granted priority review to Bristol Myers Squibb's application for Opdivo and Yervoy in first-line unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer (mCRC). The goal date is June 23, 2025.The filing was based on data from the phase 3 CheckMate-8HW trial. The combo lowered the risk of disease progression or death by 38% versus Opdivo alone in MSI-H/dMMR mCRC, according to an update of the study presented in January.The FDA has updated the label of Indivior's Sublocade for severe opioid use disorder. The new label allows healthcare providers to start treatment with Sublocade after a single dose of transmucosal buprenorphine and a one-hour observation period. This rapid initiation "may lessen some of the practical obstacles to treatment induction," according to Indivior.In addition, the FDA has approved additional injection sites for Sublocade, an extended-release formulation of buprenorphine. The drug can now be administered in the abdomen, thigh, buttock or back of the upper arm.UPDATED: Friday, Feb. 21 at 9:30 a.m. ETGilead's seladelpar has secured a conditional marketing nod in Europe to treat adults with primary biliary cholangitis (PBC).The approval covers seladelpar's use in combination with ursodeoxycholic acid (UDCA) for those who have an inadequate response to UDCA alone, or as a monotherapy for people who can't tolerate UDCA.In the U.S., seladelpar picked up FDA approval last year and is branded as Livdelzi.A chronic autoimmune disease, PCB affects roughly 22 out of 100,000 people in Europe. The disease is more common in women and causes liver damage that can progress to liver failure if untreated. Symptoms include chronic itch and fatigue, and there is no cure for the disease.The approval is largely based on results from the phase 3 Response study, which showed that roughly three times as many patients on seladelpar achieved a composite biochemical response than those on placebo. The conditional approval will be contingent on positive results from future confirmatory studies.In Japan, authorities have approved CSL's subcutaneous Andembry for the prevention of acute attacks of hereditary angioedema (HAE).The drug offers patients the first pre-filled pen presentation for monthly subcutaneous administration for long-term prophylaxis of HAE, CSL said in a press release.The Japanese nod adds to the drug's other approvals in Europe, Australia and the U.K.UPDATED: Thursday, Feb. 20 at 10:00 a.m. ETBristol Myers Squibb's Breyanzi has won reimbursement backing from England's National Institute for Health and Care Excellence (NICE) in second-line large B-cell lymphoma, becoming the first CAR-T therapy to be recommended by the agency for this cancer type. The endorsement comes after NICE had originally rejected Breyanzi in October 2024 during its draft assessment. The drug cost watchdog changed its mind after BMS "offered an improved commercial arrangement" to the NHS based on its 297,000 pound sterling (about $375,000) list price per person, according to a press release.About 600 people in England could benefit from the therapy per year, according to NICE, which called the one-time CAR-T treatment "a significant advance in lymphoma care." In another reversal, NICE has also changed a previous rejection and signed off on UCB's Fintepla as an add-on treatment for seizures associated with Lennox-Gastaut syndrome (LGS) in people ages 2 years and above. The drug already has coverage under NHS for treating seizures from Dravet syndrome.LGS is a rare but severely debilitating form of epilepsy that begins in early childhood. By NICE's estimate, about 1,400 patients live with the disease in its jurisdiction.NICE changed its mind after UCB offered "an improved discount" to the drug's list price, the agency noted. Using a cost comparison approach, UCB convinced NICE's drug reviewers that Fintepla provides similar or greater health benefits compared to cannabidiol plus clobazam—which is already covered for LGS in the NHS—at a similar or lower cost.  The drug is covered only if the frequency of drop seizures is checked every six months, at which point the frequency is reduced by at least 30% compared with before starting treatment. UPDATED: Tuesday, Feb. 18 at 11:04 a.m. ETMerck’s Welireg snagged its first two approvals in Europe, with the nods covering certain patients with von Hippel-Lidau (VHL) disease and advanced clear renal cell carcinoma (RCC).The European Commission specifically approved the medicine as a treatment for those with VHL disease who have associated, localized RCC, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors. The drug is approved in cases when localized procedures are unsuitable, making Welireg the first available treatment for VHL-associated tumors in Europe. The med also won a nod for adult patients with advanced clear cell RCC that has progressed following two or more lines of therapy, including a PD-1/L1 and at least two VEGF-targeted therapies.The European nods, which are conditional authorizations, follow similar approvals in the U.S. Merck will make the drug commercially available individual European countries after the complication of national reimbursement procedures, among other launch timing factors, the company said.Gilead’s twice-yearly pre-exposure prophylaxis (PrEP) candidate lenacapavir is on the FDA’s review table with a decision date of June 19.The drug, which is currently approved as Sunlenca for multi-drug resistant HIV, would be the first twice-yearly PrEP option on the market. The company’s FDA bid is the first step of its detailed global launch and access strategy.Gilead’s lenacapivir application is backed by two phase 3 trials, one of which showed an impressive 100% risk reduction. The company describes the med as a “foundation” for potential future HIV therapies, it said in a release.Sanofi and Regeneron’s immunology powerhouse Dupixent is aiming to secure a new use in chronic skin disease bullous pemphigoid (BP) with an FDA decision expected by June 20.If approved, the med would be the only targeted BP treatment available in the U.S. The disease is hallmarked by intense itch and blisters and impacts a population of around 27,000 adults, typically elderly adults, whose condition is not controlled by systemic corticosteroids.  The companies’ FDA bid has been granted priority review and is based on a study of 106 adults with moderate-to-severe BP. In the trial, five times more patients who received Dupixent achieved sustained disease remission compared to those on placebo, according to Sanofi. UPDATED: Friday, Feb. 14 at 9:20 a.m. ETCSL's hereditary angioedema (HAE) therapy Andembry has secured approval from the European Commission.The drug is the first once-monthly treatment targeting factor XIIa to prevent HAE attacks in patients 12 and older. In a phase 3 trial, the drug significantly reduced investigator-confirmed HAE attacks per month versus placebo, according to results published in The Lancet in April 2023.Last month, regulators in Australia and the U.K. signed off on the medicine."Andembry, CSL's first approved recombinant monoclonal antibody discovered and developed entirely by CSL, underscores our more than 40-year legacy in HAE research and treatment optimization and our decades-long journey to bring this innovation to patients," Bill Mezzanotte, M.D., CSL's head of R&D, said in a press release.Now, CSL plans to engage in access and reimbursement negotiations with national authorities across Europe as the company progresses with the rollout.Also in Europe, officials signed off on CSL and Arcturus Therapeutics' self-amplifying mRNA COVID-19 vaccine, Kostaive, for use in individuals ages 18 and older.The approval is based on results from an integrated phase 1/2/3 study and late-stage booster trials, the partners said in a press release. Late last year, the European Medicines Agency recommended the vaccine for approval.Besides Europe, the vaccine is approved in Japan.Galderma's efforts to expand the market for Nemluvio are bearing more fruit.In Europe, officials approved the subcutaneous drug for the treatment of moderate-to-severe atopic dermatitis in patients ages 12 and older. In addition, the drug scored an endorsement to treat prurigo nodularis in adults.In the late-stage Arcadia and Olympia clinical trial programs, the drug "significantly improved itch, skin lesions and sleep disturbance, in patients with moderate-to-severe atopic dermatitis and adults with prurigo nodularis, respectively," Galderma said in a Friday press release.The drug is also approved in the U.S. and is under review in Canada, Brazil, South Korea and other countries.China has approved its 22nd PD-1/L1 product. The approval went to SinoCellTech's finotonlimab for its use alongside chemo in first-line head and neck cancer. In a China phase 3 trial, the combo reduced the risk of death by 27% versus chemo alone, extending the median overall survival by 3.6 months to 14.1 months, according to results published in Nature Medicine.The go-ahead marks the first for a PD-1/L1 inhibitor that covers all head and neck cancer patients in China. In the country, Merck & Co.'s Keytruda is allowed as a monotherapy only in PD-L1-positive disease, SinoCellTech noted in a securities filing (Chinese). UPDATED: Wednesday, Feb. 12 at 10:20 a.m. ETAcoramidis, the transthyretin amyloid cardiomyopathy (ATTR-CM) drug developed by BridgeBio and licensed by Bayer in Europe, has been approved by the European Commission. Bayer plans to make the transthyretin stabilizer available in Europe under the brand name Beyonttra in the first half of this year.The approval is based on results from the phase 3 ATTRibute-CM study trial showing that Beyonttra significantly reduced the composite endpoint of all-cause mortality and cardiovascular-related hospitalizations compared with placebo. During the open-label extension phase of the study, Beyonttra demonstrated a relative risk reduction in deaths of 34% after 42 months.Beyonttra's launch is one of Bayer's “must-win battles” as the German company navigates the loss of exclusivity of its top-selling drug, blood thinner Xarelto, Stefan Oelrich, head of Bayer’s pharma division, said at the J.P. Morgan Healthcare Conference in January.Travere Therapeutics said it has aligned with the FDA on its plan to submit Filspari for traditional approval in focal segmental glomerulosclerosis (FSGS), a rare kidney disease. The move comes after a Filspari phase 3 trial in that indication failed on its primary endpoint of eGFR slope, a measurement of kidney disease progression. It also comes as findings from a collaborative global project called ARASOL linked a decrease in proteinuria over 24 months to a reduction in the risk of kidney failure in FSGS. Filspari has shown a benefit in lowering proteinuria. Travere plans to file this application around the end of March using existing data from the phase 3 DUPLEX and phase 2 DUET studies. The company will request a priority review, which Leerink Partners analysts believe is likely to be granted as there are no approved therapies for FSGS. Given that the understanding around proteinuria as a potential full approval endpoint is new, Leerink analysts said it would not be surprising if the FDA wanted to discuss the results during an advisory committee meeting.An approval in FSGS would be "transformational" for Filspari and Travere, Leerink said in a Tuesday note, because it could give the drug $1.1 billion in peak sales across the U.S. and EU based on a "relatively conservative peak penetration" of less than 20%. For Filspari sales, the FSGS indication could eventually surpass IgA nephropathy, for which Filspari is currently approved and is starting to face more competition, according to Leerink Partners.UPDATED: Monday, Feb. 10 at 9:45 a.m. ETBristol Myers Squibb’s cancer combo Opdivo plus Yervoy has scored an approval recommendation in the E.U., thanks to an endorsement from the EMA’s Committee for Medicinal Products for Human Use (CHMP) as a first-line treatment in adults with unresectable or advanced hepatocellular carcinoma.With the thumbs up from CHMP, the Opvido-Yervoy combo now moves forward to the European Commission (EC) for a final approval decision.The CHMP based its green light on results from the combo’s phase 3 CheckMate -9DW trial, in which patients on Opdivo and Yervoy charted a median overall survival of 23.7 months, compared to 20.6 months in patients who received an alternative regimen of Lenvima (lenvatinib) or sorafenib.BMS’ cancer combo is up for a similar approval in the U.S., with the FDA scheduled to make its decision on a potential first-line unresectable HCC nod by April 21.The drugmaker also won a CHMP endorsement for its CAR-T therapy Breyanzi to treat adults with relapsed or refractory follicular lymphoma (FL) who’ve previously tried two or more systemic therapies.BMS used data from its phase 2 TRANSCEND FL study to support the recommendation. In the trial, Breyanzi charted an overall response rate of 97.1% and a complete response rate of 94.2%, which was sufficient to meet the study’s primary and secondary endpoints, BMS said in a release.Breyanzi already carries multiple oncology approvals in Europe, including in relapsed or refractory diffuse large B-cell lymphoma and high grade B-cell lymphoma.Late last month, the FDA gave a thumbs up to German drugmaker medac’s alkylating agent Grafapex, also known as treosulfan, alongside the antimetabolite medicine fludarabine, as a preparative regimen for allogeneic hematopoietic stem cell transplantation (alloHSCT) in adults and kids ages one and older with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).Alkylating agents cause breakage of DNA strand to help thwart the ability of cancer cells to multiply.Medac’s approval was backed up by a study looking primarily at overall survival in 570 adults ages 18 to 70 years old with AML or MDS who were randomized to receive either Grafapex or busulfan with fludarabine as a preparative regimen for allogeneic transplant. UPDATED: Wednesday, Feb. 5 at 9:05 a.m. ETNovartis' Kisqali has secured a label expansion from the U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA).The new approval covers Kisqali's use in combination with an aromatase inhibitor (AI) in patients with HR+/HER2- early breast cancer at high risk of recurrence. Previously, the drug was approved by the MHRA to treat advanced breast cancer, according to a Feb. 4 press release from Novartis.The latest approval is based on results from the phase 3 NATALEE trial, which showed that the addition of Kisqali to treatment with an AI cut the risk of invasive disease, recurrence, or death compared to AI treatment alone in patients with HR+/HER2- early breast cancer.Last fall, the drug picked up an FDA nod in patients with HR+/HER2- early breast cancer based on the NATALEE study results.Vaccine specialist Valneva has picked up an approval from the MHRA, as well.The agency approved the company's chikungunya vaccine, Ixchiq, for use in people 18 years of age and older. Late-stage trial data show the vaccine "induces a rapid and strong immune response," according to a press release from the company.Valneva manufactures the shot at its facility in Livingston, Scotland.Previously, the vaccine secured approvals in the United States, Canada and Europe. UPDATED: Friday, Jan. 31 at 9:35 a.m. ETVertex's groundbreaking launch of Casgevy keeps racking up more milestones around the globe.Friday, the company said it has reached a reimbursement agreement with NHS England that'll allow eligible patients with sickle cell disease access to the CRISPR/Cas9 gene-edited therapy. Along with the NHS England agreement, the National Institute for Health and Care Excellence (NICE) has signed off on the drug's use, Vertex said in a press release.The reimbursement deal follows a similar agreement, unveiled last summer, for the drug in patients with transfusion-dependent beta thalassemia.“We are pleased to have reached this new agreement that ensures both eligible SCD and TDT patients can now be treated with Casgevy, recognizing the value a one-time treatment can provide to patients, their families and the healthcare system," Vertex International senior vice president Ludovic Fenaux said in a statement.In its approval bid for Fabhalta in C3 glomerulopathy, Novartis will no longer face an FDA advisory committee meeting, CEO Vas Narasimhan said on Friday.The advisory committee meeting had been scheduled for next month but will no longer take place.Speaking with members of the media on Friday, Narasimhan said the "FDA no longer deemed it necessary to hold an advisory committee meeting.""We think it's based on, you know, the overall data package we've been able to generate, and the additional follow on data that we've provided the agency," the CEO continued. So we look forward to now bringing forward that medicine as a third indication approval for Fabhalta."Amid Europe's ongoing regulatory review of Eisai and Biogen's Alzheimer's disease medicine Leqembi, the European Commission (EC) has flagged new safety information for review.In November, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended market approval of the drug. Since then, EC has asked the committee to "consider information on the safety of lecanemab that became available after the adoption of the CHMP opinion," the partners said in a Friday press release. The companies didn't reveal the nature of the new safety information flagged by the EC. CHMP will dig into the new info during a meeting next month, according to the release."We believe that the EC’s requests can be addressed with existing information and will be evaluated by the CHMP because of the clear and sufficient information available," the partners said in the release. "We will continue to work closely with the authorities toward approval in the EU."UPDATED: Monday, Jan. 27 at 10:43 a.m. ETMerck’s Welireg is up for a potential FDA label expansion by May 26. The agency granted a priority review to the company's bid to expand the drug's label into advanced, unresectable, or metastatic pheochromocytoma and paraganglioma (PPGL), which would add to currently approved indications in advanced renal cell carcinoma and von Hippel-Lindau disease.Welireg generated $139 million in sales during 2024’s third quarter and is the only approved HIF-2α inhibitor in the U.S.Merck’s application in PPGL is based on objective response rate and duration of response rate data from the phase 2 LITESPARK-015 trial, which enrolled 322 patients and will be presented at an upcoming medical meeting, according to the company. Up to 2,000 new cases of the tumors are diagnosed yearly in the U.S.Merck is evaluating Welireg across other rare oncologic diseases both on its own and in combination with other therapies. The FDA is evaluating Alvotech and Teva’s proposed biosimilar to Johnson & Johnson’s Simponi and Simponi Aria.Alvotech, which is responsible for development and manufacturing under its longstanding collaboration with Teva, announced positive top line results from its confirmatory trial of the Simponi copycat back in April. The partners expect the FDA to complete its review process in the fourth quarter of 2025. Simponi was first approved in 2009 and treats a variety of inflammatory conditions including rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis, among others. Simponi is an injected drug, while Simponi Aria is the infused version. The franchise picked up $2.19 billion for J&J in 2023 sales.Alvotech and Teva's product is the first biosimilar to the blockbuster arthritis med to be accepted for review.UPDATED: Thursday, Jan. 23 at 10:00 a.m. ETBiogen said its applications for a high-dose version of the spinal muscular atrophy med Spinraza have been accepted for review by the FDA and the European Medicines Agency. The new regimen comprises a more rapid loading regimen of two 50 mg doses given two weeks apart and a maintenance regimen given at 28 mg every four months. Currently, the drug's FDA-approved loading regimen features three doses given 14 days apart, plus a fourth dose 30 days after the third. The drug is currently approved in 12 mg doses.The high-dose applications are based on results from the phase 2/3 Devote trial, which favored the high-dose version versus an external sham control and the currently available dose on the measure of patients' reduction in plasma neurofilament light chain (NfL), a marker for neurodegeneration.The higher dose regimen also reduced the risk of death or permanent ventilation by 68% relative to sham and 30% compared with the 12 mg regimen.The high-dose Spinraza is Biogen's answer to competitors such as Novartis' gene therapy Zolgensma.Amneal has won FDA approvals for generics referencing AbbVie's Alzheimer's combo drug Namzaric and Novartis' cancer therapy Afinitor Disperz. Amneal has launched the Namzaric generic with 180-day market exclusivity.Separately, Amneal said it has won tentative approval from the FDA for a generic to Bausch Health's star irritable bowel syndrome med Xifaxan. Bausch Health has previously reached settlement agreements with Teva, Sun Pharma and Sandoz, pushing their generic entry dates into 2028 or upon an earlier launch of a rival generic product. Xifaxan is one of 15 drugs up for the second cycle of Medicare price negotiations under the Inflation Reduction Act. UPDATED: Tuesday, Jan. 21 at 8:57 a.m. ETThe European Commission has approved Johnson & Johnson's combination of Lazcluze and Rybrevant for the first-line treatment of certain adults with advanced non-small cell lung cancer (NSCLC), the company said in a press release.Specifically, the combo is approved in Europe for patients with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R (L858R) substitution mutations.In approving the use, EU regulators reviewed results from the phase 3 MARIPOSA study. The trial showed that the combination reduced the risk of disease progression or death by 30% compared with AstraZeneca's Tagrisso in patients with newly diagnosed, EGFR-mutant non-small cell lung cancer. The results yielded a U.S. approval for the J&J combination last summer. Earlier this month, J&J touted an overall survival win from the study. At the time, a company executive heralded the regimen as "a new standard of care for patients with EGFR non-small cell lung cancer."Also in Europe, officials have endorsed GSK's Jemperli to treat all adult patients with primary advanced or recurrent endometrial cancer, including those with mismatch repair proficient (MMRp)/microsatellite stable (MSS) tumors. MMRp and MSS cases represent approximately 75% of patients diagnosed with endometrial cancer, GSK said in a press release.The approval covers Jemperli's use in combination with chemotherapy.Late last year, Europe's Committee for Medicinal Products for Human Use (CHMP) recommended the label expansion.In England, the National Institute for Health and Care Excellence (NICE) recommended AstraZeneca's Tagrisso for treating people with EGFR mutation-positive non-small-cell lung cancer (NSCLC) after surgery.The recommendation means that hundreds of patients will benefit from routine access to the drug, which is taken daily alongside chemotherapy, according to a notice from the Roy Castle Lung Cancer Foundation.Previously, EGFR mutation-positive NSCLC patients relied on chemotherapy to prevent cancer recurrence after surgery, according to the foundation.“I am pleased we have been able to recommend that this targeted treatment for a specific gene mutation of lung cancer will be routinely available on the NHS," NICE's director of medicines evaluation, Helen Knight, said in a statement.UPDATED: Thursday, Jan. 16 at 10:25 a.m. ETEli Lilly's Omvoh has picked up its second major FDA nod. After the IL-23 inhibitor's approval in 2023 to treat ulcerative colitis, the agency has now signed off on Omvoh to treat Crohn's disease. Specifically, the FDA approved Omvoh to treat moderately to severely active Crohn's disease in adults.In a phase 3 study, 53% of patients treated with Omvoh achieved clinical remission after a year of treatment. That number compared with 36% for those on placebo.In addition, 46% of patients treated with Omvoh had visible healing of the intestinal lining after a year. In the placebo group, the number was 23%.The trial enrolled patients with moderately to severely active Crohn's disease who had experienced an inadequate response, loss of response or who could not tolerate corticosteroids, immunomodulators and/or biologics.With the FDA nod, Lilly's drug will go up against some heavy hitters in the Crohn's space, including Johnson & Johnson's Stelara and AbbVie's duo of Skyrizi and Rinvoq.UPDATED: Wednesday, Jan. 15 at 8:12 a.m. ETSanofi's Sarclisa has gained approval from China's National Medical Products Administration (NMPA) to treat adults with multiple myeloma who've received at least one prior line of therapy, including lenalidomide and a proteasome inhibitor.The approval covers Sarclisa's use in combination with pomalidomide and dexamethasone (Pd).Regulators in China endorsed the drug based on results from the phase 3 ICARIA-MM study, as well as bridging data from a China-based study, Sanofi said in a press release. In ICARIA-MM, the addition of Sarclisa to Pd resulted in a 40% reduction in patients' risk of disease progression or death. "We look forward to continuing to build strong partnerships with the medical community, local companies, and authorities in China as we work to bring more innovative treatments to patients," Sanofi's head of oncology, Olivier Nataf, said in a statement.As Biogen and Eisai work to grow Leqembi in Alzheimer's disease, the FDA has accepted a biologics license application for the drug's subcutaneous maintenance dosing option. With a potential approval, Leqembi would become the first anti-amyloid therapy to offer at-home maintenance dosing.The FDA is set to make a decision on the filing by August 31, according to a Biogen press release.UPDATED: Thursday, Jan. 9 at 11:15 a.m. ETEngland’s National Institute for Health and Care Excellence (NICE) recommended AstraZeneca’s Lynparza for use in the region’s National Health Service, clearing the way for around 1,200 eligible patients to reap the benefits of the treatment.The recommendation covers Lynparza’s use in patients with HER2-negative, locally advanced or metastatic breast cancer who have BRCA1 or BRCA2 mutations and have had chemotherapy. NICE based its decision in part on results from the company’s OlympiAD phase 3 study, which proved that the drug could cut the risk of disease worsening or death by 42%.NICE had originally begun its assessment of the treatment in that indication in 2018, but ended it citing a lack of evidence submitted by the company. This time around, NICE assessed the med using a faster cost comparison process that compared Lynparza to Pfizer’s Talzenna in benefits and cost. Stallergenes Greer’s peanut allergy treatment Palforzia scored a label expansion in Europe, extending the drug’s use to toddlers aged 1 through 3 years old.Palforzia is now cleared for use in those aged 1 to 17 years old and is the only approved oral immunotherapy in the U.S. and Europe for toddlers with a confirmed peanut allergy diagnosis. The drug is meant to gradually increase the body’s ability to tolerate small amounts of peanuts through carefully controlled initial dose escalation and up-dosing.The European Commission based its nod on the company’s POSEIDON phase 3 study, which was a double-blind, placebo-controlled food challenge after six months of treatment. Agios Pharmaceuticals’ Pyrukynd is up for an FDA decision in non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia on Sept. 7.The company filed its bid for approval last month after finding success in two phase 3 trials assessing the drug in the non-transfusion-dependent and transfusion-dependent populations. Thalassemia is a rare, inherited blood disorder with limited treatment options. The disease is hallmarked by the abnormal production of hemoglobin, which causes chronic anemia and can lead to other complications.Pyruknd won its first approval in 2022 for pyruvate kinase (PK) deficiency, marking the first drug approved to treat the disease. Merck’s Gardasil HPV vaccine won approval from the National Medical Products Administration of China for males aged 9 to 26 years old, making the first option of its kind available for the country's male population.The vaccine was previously approved for use in females who are 9 to 45 years of age and has helped protect over 50 million women in China from certain HPV-related cancers and diseases since its first approval, Merck’s president of Human Health International, Joseph Romanelli, said in a release.Gardasil has recently seen slumping sales in China, where it's being marketed by Merck’s regional commercial partner Zhifei Biological Products. Rob David previously called the expansion to the male population a “significant opportunity.” UPDATED: Tuesday, Jan. 7 at 3:57 p.m. ETThe FDA tacked on additional warning information to the prescribing information for Agios’ pyruvate kinase (PK) deficiency drug Pyrukynd, flagging instances of liver deficiency in certain situations.The label update follows liver injury observations in patients who received Pyrukynd at a dose higher than is recommended for PK deficiency, according to an SEC filing from the company.The drug was approved by the FDA in 2022 as the first medicine to treat hemolytic anemia in adults with PK deficiency. Besides this use, the company is exploring expansions into beta thalassemia and sickle cell disease.The updated "Warnings and Precautions" section urges prescribers to run liver tests on patients before initiating treatment and monthly for the first six months of treatment. Patients should stop treatment if hepatic injury is suspected.Bayer is vying for a third indication for its Nubeqa (darolutamide) in China, this time for its use in combination with androgen deprivation therapy (ADT) in patients with metastatic hormone-sensitive prostate cancer.The company filed an application for the label expansion with China’s National Medical Products Administration, leveraging data from its phase 3 ARANOTE trial. In the study, Nubeqa and ADT significantly reduced the risk of radiological progression or death by 46% compared with placebo plus ADT.The nod would add to the drug’s two existing approvals in China, which cover other prostate cancer patient populations. It is estimated that the prevalence of prostate cancer in China will exceed 161,000 cases annually by 2026, according to Bayer. Dizal’s non-small cell lung cancer (NSCLC) candidate sunvozertinib will get a speedy FDA review after the agency granted priority review to the company’s new drug application.The company is specifically going after an approval to treat locally advanced or metastatic non-small cell lung cancer (NSCLC) in patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy. Sunvozertinib won its world-first approval in China in 2023. UPDATED: Monday, Jan. 6 at 10:25 a.m. ETAmgen's DLL3-targeted bispecific antibody Imdylltra, known as Imdelltra in the U.S., has won a conditional marketing authorization from the U.K.'s Medicines and Healthcare products Regulatory Agency as a third-line therapy for small cell lung cancer.The conditional nod is based on results from the phase 2 DeLLphi-301 trial, which showed that the T-cell engager triggered a tumor response rate of 41%, with the median response lasting 9.7 months. It follows an FDA approval in May 2024.China has approved Astellas' first-in-class Claudin 18.2 drug Vyloy, following similar moves by drug regulators in Japan and the U.S.The drug's Chinese indication is in first-line, CLDN18.2-positive, HER2-negative advanced gastric or gastroesophageal junction adenocarcinoma. The CLDN18.2 biomarker is expressed by about 35% of Chinese stomach cancer patients, according to Astellas.The approval was backed by two global phase 3 trials, dubbed Glow and Spotlight, which evaluated Vyloy in combination with the chemotherapy regimens CAPOX and mFOLFOX6, respectively. Both trials linked the drug's chemo combo to longer life expectancy and progression-free survival time compared with chemo alone.UPDATED: Friday, Jan. 3 at 2:45 p.m. ETGSK’s IL-5 inhibitor Nucala scored a nod in China to treat people with inadequately controlled chronic rhinosinusitis with nasal polyps (CRSwNP), a condition that impacts nearly 30 million people in China.The drug was approved by China’s National Medical Products Administration for use as an add-on therapy along with intranasal corticosteroids for patients with CRSwNP who don’t achieve adequate disease control with systemic corticosteroids or surgery. The indication is Nucala’s third in China and comes after prior approvals in eosinophilic asthma and eosinophilic granulomatosis with polyangiitis.An FDA advisory committee panel is scheduled to discuss the merits of Novartis’ oral factor B inhibitor Fabhalta in the rare kidney disease C3 glomerulopathy (C3G).The Cardiovascular and Renal Drugs Advisory Committee will meet on February 24, according to an FDA document (PDF). The drug was approved by the agency in 2023 as a treatment for paroxysmal nocturnal hemoglobinuria, winning the title of the first oral monotherapy approved for the rare blood disorder. Fabhalta received a second nod for immunoglobulin A nephropathy (IgAN) in August.In studies, Fabhalta proved that it could reduce proteinuria for C3G patients, a marker for delaying progression to kidney failure. So far, there are no approved treatments for the ultra-rare disease, which causes about 50% of patients to progress to kidney failure within ten years of diagnosis. UPDATED: Thursday, Jan. 2 at 1:45 p.m. ETShortly before the new year, Merck & Co. scored an approval for its potential blockbuster Winrevair in the U.K. The pulmonary arterial hypertension (PAH) drug, which also goes by the name sotatercept, was originally picked up via Merck’s $11.5 billion acquisition of Acceleron back in 2021.The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) approved Winrevair in combination with other PAH therapies to improve exercise capacity in adult patients who live with moderate or marked limitations of physical activity.Patients with PAH suffer from high blood pressure in the blood vessels that supply the lungs, which can ultimately damage the right side of the heart. Winrevair, for its part, targets the causes of PAH responsible for the narrowing of the arteries in the lungs, in turn making it easier for the heart to pump blood to the pair of organs, according to the U.K. government’s release.The MHRA based its approval on a study of 323 PAH patients that saw Winrevair best placebo at improving exercise ability. The main measure of effectiveness in the trial was the difference in distance patients could walk in six minutes before and after treatment.At the trial’s 24-week treatment mark, patients on Winrevair plus other PAH medicines improved their walking distance by around 34 meters, versus one meter in patients who received placebo.Merck’s U.K. nod for Winrevair follows green lights from the U.S. FDA last March and the European Commission in August.At the time of the drug’s U.S. approval last year, J.P. Morgan’s Chris Schott estimated Winrevair could eventually reel in peak sales between $3 billion and $4 billion. 

Dosing ongoing in the Phase 2 pivotal study of RP-A501 for Danon disease; Program update expected in the first half of 2025 New England Journal of Medicine publication of Phase 1 study of RP-A501 to treat patients with Danon disease Long-term data from the Phase 1 trial of RP-A501 for Danon disease presented at AHA on safety and meaningful efficacy Enrollment completed in low dose cohort of Phase 1 study of RP-A601 for PKP2-ACM; Initial data expected in the first half of 2025 Regulatory reviews ongoing for KRESLADI (severe LAD-I) and RP-L102 (Fanconi Anemia) Cash, cash equivalents and investments of approximately $372.3M ; expected operational runway into the third quarter of 2026 CRANBURY, N.J. --(BUSINESS WIRE)--Feb. 27, 2025-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the fourth quarter and year ended December 31, 2024 . “In 2024, we made strong progress in advancing our gene therapy pipeline, underscored by the New England Journal of Medicine publication of the Phase 1 study of RP-A501 for Danon disease and long-term data presented at AHA showing its safety and meaningful efficacy up to five years. Our momentum continues as we progress with the Phase 2 pivotal trial of RP-A501 and the Phase 1 trial of RP-A601 for PKP2-ACM, and we remain on track to submit the IND for BAG3-DCM in the first half of 2025,” said Gaurav Shah , M.D., Chief Executive Officer of Rocket Pharmaceuticals . “Looking ahead to 2025, we will maintain our focus and resources on advancing our AAV cardiovascular programs while seeking to realize value in our full pipeline in a thoughtful manner, so we deliver the greatest value to our patients and shareholders.” Recent Pipeline and Operational Updates Dosing in the Phase 2 pivotal study of RP-A501 for Danon disease is ongoing. Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Program update anticipated in the first half of 2025. Long-term data from the Phase 1 study of RP-A501 for Danon disease published in The New England Journal of Medicine and new data presented at the American Heart Association’s 2024 Late-Breaking Science sessions. RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years). Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months. All evaluable patients had reductions in NYHA heart failure (from Class II to Class I; no longer displaying symptoms of heart failure), improvements in KCCQ (median 27-point increase), and substantial improvements in troponin (median reduction 84%) and BNP (median reduction 57%) observed 24-54 months after treatment. Progressed the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM). Completed enrollment of all patients in the low dose cohort, and initial data from the Phase 1 study is expected in the first half of 2025. Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe , representing the largest market opportunity in Rocket’s pipeline of disclosed programs. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412. Progressed BAG3-associated dilated cardiomyopathy preclinical program. Nonclinical, IND-enabling studies are ongoing. Submission of the IND is anticipated in the first half of 2025. Progressed RP-L102 investigational gene therapy for Fanconi Anemia (FA). Initiated rolling Biologics License Application (BLA) for RP-L102 and submission of the final module is anticipated in 2025. Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is ongoing. U.S. Food and Drug Administration (FDA) review of limited additional Chemistry Manufacturing and Controls (CMC) information ongoing for KRESLADITM (marnetegragene autotemcel; marne-cel) for the treatment of severe leukocyte adhesion deficiency-I (LAD-I). Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I. The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research . Submission of complete BLA to resolve Complete Response Letter anticipated in 2025. Raised net proceeds of $182.5M through public offering. On December 11, 2024 , the Company completed a public offering of approximately 15.2 million shares of our common stock at a public offering price of $12.50 per share and pre-funded warrants to purchase 0.4 million shares of common stock at a price of $12.49 per warrant. The gross proceeds to Rocket from the public offering were approximately $194.7 million , net of $12.2 million of offering costs, commissions, legal and other expenses for net proceeds of $182.5 million . Details of the Phase 2 pivotal study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034. Program update anticipated in the first half of 2025. RP-A501 demonstrated safety and meaningful efficacy; all evaluable patients show cardiac LAMP2 expression and ≥10% reduction in LV mass index at 12 months and sustained through most recent follow up (up to five years). Evidence of sustained clinically meaningful improvement was observed in pediatric patients followed up to 24 months and adult/adolescent patients followed up to 60 months. All evaluable patients had reductions in NYHA heart failure (from Class II to Class I; no longer displaying symptoms of heart failure), improvements in KCCQ (median 27-point increase), and substantial improvements in troponin (median reduction 84%) and BNP (median reduction 57%) observed 24-54 months after treatment. Completed enrollment of all patients in the low dose cohort, and initial data from the Phase 1 study is expected in the first half of 2025. Ongoing internal estimates confirm that PKP2-ACM affects approximately 50,000 people in the U.S. and Europe , representing the largest market opportunity in Rocket’s pipeline of disclosed programs. Details of the Phase 1 study can be found at www.ClinicalTrials.gov under the NCT identifier NCT05885412. Nonclinical, IND-enabling studies are ongoing. Submission of the IND is anticipated in the first half of 2025. Initiated rolling Biologics License Application (BLA) for RP-L102 and submission of the final module is anticipated in 2025. Regulatory review for RP-L102 for the treatment of FA by the European Medicines Authority is ongoing. Rocket previously disclosed that the FDA requested limited additional CMC information to complete its review of KRESLADI to treat severe LAD-I. The Company continues to work with senior leaders and reviewers from the FDA’s Center for Biologics Evaluation and Research . Submission of complete BLA to resolve Complete Response Letter anticipated in 2025. On December 11, 2024 , the Company completed a public offering of approximately 15.2 million shares of our common stock at a public offering price of $12.50 per share and pre-funded warrants to purchase 0.4 million shares of common stock at a price of $12.49 per warrant. The gross proceeds to Rocket from the public offering were approximately $194.7 million , net of $12.2 million of offering costs, commissions, legal and other expenses for net proceeds of $182.5 million . Fourth Quarter and Full Year 2024 Financial Results Cash position. Cash, cash equivalents and investments as of December 31, 2024 , were $372.3 million . R&D expenses. Research and development expenses were $171.2 million for the twelve months ended December 31, 2024 , compared to $186.3 million for the twelve months ended December 31, 2023 . The decrease of $15.1 million in R&D expenses was primarily driven by decreases in manufacturing and development and direct material costs of $19.9 million . The decreases were partially offset by increases in the costs for professional fees and consultants of $4.0 million , non-cash stock compensation expense of $1.3 million and depreciation expense of $2.2 million . G&A expenses. General and administrative expenses were $102.0 million for the twelve months ended December 31, 2024 , compared to $73.3 million for the twelve months ended December 31, 2023 . The increase in G&A expenses was primarily driven by increases in commercial preparation related expenses which consisted of commercial strategy, medical affairs, market development and pricing analysis expenses of $17.6 million , legal expenses of $4.8 million , non-cash stock compensation expense of $3.2 million , and compensation and benefit expense of $2.1 million . Net loss. Net loss was $258.7 million or $2.73 per share (basic and diluted) for the twelve months ended December 31, 2024 , compared to $245.6 million or $2.92 (basic and diluted) for the twelve months ended December 31, 2023 . Shares outstanding. 106,453,818 shares of common stock were outstanding as of December 31, 2024 . Financial Guidance Cash position. As of December 31, 2024 , Rocket had cash, cash equivalents and investments of $372.3 million . Rocket expects such resources will be sufficient to fund its operations into the third quarter of 2026, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs. About Rocket Pharmaceuticals, Inc. Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of investigational genetic therapies designed to correct the root cause of complex and rare disorders. Rocket’s innovative multi-platform approach allows us to design the optimal gene therapy for each indication, creating potentially transformative options that enable people living with devastating rare diseases to experience long and full lives. Rocket’s adeno-associated viral (AAV) vector-based cardiovascular portfolio includes a late-stage program for Danon Disease, a devastating heart failure condition resulting in thickening of the heart, an early-stage program in clinical trials for PKP2-arrhythmogenic cardiomyopathy (ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and a pre-clinical program targeting BAG3-associated dilated cardiomyopathy (DCM), a heart failure condition that causes enlarged ventricles. Rocket’s lentiviral (LV) vector-based hematology portfolio consists of late-stage programs for Fanconi Anemia (FA), a difficult-to-treat genetic disease that leads to bone marrow failure (BMF) and potentially cancer, Leukocyte Adhesion Deficiency-I (LAD-I), a severe pediatric genetic disorder that causes recurrent and life-threatening infections which are frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia. For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X. Rocket Cautionary Statement Regarding Forward-Looking Statements This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical facts contained in this release are forward-looking statements. You should not place reliance on these forward-looking statements, which often include words such as “could,” “believe,” “expect,” “anticipate,” “intend,” “plan,” “will give,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms. These forward-looking statements include, but are not limited to, statements concerning Rocket’s expectations regarding the safety and effectiveness of product candidates that Rocket is developing to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon Disease (DD) and other diseases, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, the expected timing and outcome of Rocket’s regulatory interactions and planned submissions, including the timing and outcome of the FDA’s review of the additional CMC information that Rocket will provide in response to the FDA’s request, the safety, effectiveness and timing of pre-clinical studies and clinical trials, Rocket’s ability to establish key collaborations and vendor relationships for its product candidates, Rocket’s ability to develop sales and marketing capabilities or enter into agreements with third parties to sell and market its product candidates, Rocket’s ability to expand its pipeline to target additional indications that are compatible with its gene therapy technologies, Rocket’s ability to transition to a commercial stage pharmaceutical company, and Rocket’s expectation that its cash, cash equivalents and investments will be sufficient to funds its operations into the third quarter of 2026. Although Rocket believes that the expectations reflected in the forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially from those indicated by these forward-looking statements as a result of various important factors, including, without limitation, Rocket’s dependence on third parties for development, manufacture, marketing, sales and distribution of product candidates, the outcome of litigation, unexpected expenditures, Rocket’s competitors’ activities, including decisions as to the timing of competing product launches, pricing and discounting, Rocket’s ability to develop, acquire and advance product candidates into, enroll a sufficient number of patients into, and successfully complete, clinical studies, the integration of new executive team members and the effectiveness of the newly configured corporate leadership team, Rocket’s ability to acquire additional businesses, form strategic alliances or create joint ventures and its ability to realize the benefit of such acquisitions, alliances or joint ventures, Rocket’s ability to obtain and enforce patents to protect its product candidates, and its ability to successfully defend against unforeseen third-party infringement claims, as well as those risks more fully discussed in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2024 , filed February 27, 2025 with the SEC and subsequent filings with the SEC including our Quarterly Reports on Form 10-Q. Accordingly, you should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise. 2024 2023 2024 2023 $ 37,357 $ 41,744 $ 171,244 $ 186,342 25,337 21,535 101,961 73,317 62,694 63,279 273,205 259,659 (62,694 ) (63,279 ) (273,205 ) (259,659 ) $ (473 ) (470 ) (1,886 ) (1,875 ) 1,617 814 8,267 5,288 $ 1,223 3,275 8,078 10,651 $ (60,327 ) $ (59,660 ) $ (258,746 ) $ (245,595 ) $ (0.62 ) $ (0.64 ) $ (2.73 ) $ (2.92 ) 97,530,032 93,336,541 94,807,773 84,009,004 $ 372,336 $ 407,495 527,700 566,341 64,466 73,767 463,234 492,574 View source version on businesswire.com: https://www.businesswire.com/news/home/20250227281067/en/ Investors Meg Dodge mdodge@rocketpharma.com Media Kevin Giordano media@rocketpharma.com Source: Rocket Pharmaceuticals, Inc.