Fruquintinib

— US$20 million payment based on sales of FRUZAQLA® in metastatic colorectal cancer —HONG KONG and SHANGHAI and FLORHAM PARK, N.J., Oct. 31, 2024 (GLOBE NEWSWIRE) -- HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM:​HCM; HKEX:​13) today announces that it will receive a US$20 million milestone payment from its partner Takeda (TSE:​4502/​NYSE:​TAK), triggered by reaching over US$200 million in sales of FRUZAQLA® (fruquintinib) for metastatic colorectal cancer (“CRC”). CRC is the second most common cause of cancer-related deaths in the US. There are approximately 840,000 new cases of CRC each year across the US, Europe and Japan. Takeda delivered US$203 million in net sales of FRUZAQLA® in the nine months ended September 2024. This US$20 million payment will be the first ever commercial milestone payment received by HUTCHMED. Takeda has the exclusive worldwide license to further develop, commercialize, and manufacture fruquintinib outside of mainland China, Hong Kong and Macau, and markets under the FRUZAQLA® brand name. It received approval in the US in November 2023, in the EU in June 2024, in Switzerland in August 2024, in Canada, Japan and the United Kingdom in September 2024 and in Argentina, Australia and Singapore in October 2024. Regulatory applications are progressing in many other jurisdictions. “The achievement of US$200 million in sales is a testament to Takeda’s commercial strength in launching global brands, the clinical benefit of fruquintinib and the success of our partnership strategy for commercializing our medicines outside of China,” said Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED. “Receiving the US$20 million milestone payment will strengthen our balance sheet as we look to expand the use of fruquintinib into new indications, and highlights our strategy of global partnerships across our broad pipeline.” About CRC CRC is a cancer that starts in either the colon or rectum. According to the International Agency for Research on Cancer/World Health Organization, CRC is the third most prevalent cancer worldwide, associated with more than 1.9 million new cases and 900,000 deaths in 2022. In the US, it is estimated that 153,000 patients will be diagnosed with CRC and 53,000 deaths from the disease will occur in 2024.1 In Europe, CRC was the second most common cancer in 2022, with approximately 538,000 new cases and 248,000 deaths. In Japan, CRC was the most common cancer, with an estimated 146,000 new cases and 60,000 deaths, in 2022.2,3 Although early-stage CRC can be surgically resected, metastatic CRC remains an area of high unmet need with poor outcomes and limited treatment options. Some patients with metastatic CRC may benefit from personalized therapeutic strategies based on molecular characteristics; however, most patients have tumors that do not harbor actionable mutations.4,5,6,7,8 About Fruquintinib Approvals Global regulatory submissions are based on data from two large, randomized, controlled Phase III trials, the global, multi-regional FRESCO-2 trial and the FRESCO trial conducted in China, showing consistent benefit among a total of 734 patients treated with fruquintinib. Safety profiles were consistent across trials. Results from the FRESCO-2 trial were published in The Lancet in June 2023,9 while results from the FRESCO trial were published in The Journal of the American Medical Association, JAMA.10 In mainland China, Hong Kong and Macau, fruquintinib is co-marketed by HUTCHMED and Eli Lilly and Company under the brand name ELUNATE®. It was included in the China National Reimbursement Drug List (NRDL) in January 2020. Since its launch in China, over 100,000 patients with colorectal cancer have been treated with fruquintinib. About Fruquintinib Fruquintinib is a selective oral inhibitor of all three VEGF receptors (VEGFR-1, -2 and -3). VEGFR inhibitors play a pivotal role in inhibiting tumor angiogenesis. Fruquintinib was designed to have enhanced selectivity that limits off-target kinase activity, allowing for drug exposure that achieves sustained target inhibition and flexibility for potential use as part of a combination therapy. About HUTCHMED HUTCHMED (Nasdaq/AIM:​HCM; HKEX:​13) is an innovative, commercial-stage, biopharmaceutical company. It is committed to the discovery and global development and commercialization of targeted therapies and immunotherapies for the treatment of cancer and immunological diseases. It has approximately 5,000 personnel across all its companies, at the center of which is a team of about 1,800 in oncology/​immunology. Since inception it has focused on bringing cancer drug candidates from in-house discovery to patients around the world, with its first three medicines marketed in China, the first of which is also approved in the US, Europe and Japan. For more information, please visit www.hutch-med.com or follow us on LinkedIn. E.U. IMPORTANT SAFETY INFORMATION Please consult the FRUZAQLA (fruquintinib) Summary of Product Characteristics (SmPC) before prescribing. Guidance for use: FRUZAQLA should be initiated by a physician experienced in the administration of anticancer therapy. Patients should be given the package leaflet. CONTRAINDICATIONS: Hypersensitivity to the active substance or to any of the excipients. SPECIAL POPULATIONS: Renal impairment: No dose adjustment is required for patients with mild, moderate, or severe renal impairment; Hepatic impairment: No dose adjustment is required for patients with mild or moderate hepatic impairment. FRUZAQLA is not recommended for use in patients with severe hepatic impairment as FRUZAQLA has not been studied in this population; Elderly: No dose adjustment is required in patients aged 65 years or above; Pediatric population: There is no relevant use of FRUZAQLA in the pediatric population for the indication of metastatic colorectal cancer; Women of childbearing potential / Contraception in females: Women of childbearing potential should be advised to use highly effective contraception during treatment and for at least 2 weeks following the last dose of FRUZAQLA; Pregnancy: There are no clinical data available on the use of FRUZAQLA in pregnant women. Based on its mechanism of action, FRUZAQLA has the potential to cause fetal harm. Animal studies have shown reproductive toxicity, including fetal malformations. FRUZAQLA should not be used during pregnancy unless the clinical condition of the woman requires treatment with FRUZAQLA. If FRUZAQLA is used during pregnancy or if the patient becomes pregnant while on treatment, the patient must be informed of the potential hazard to the fetus; Breast-feeding: The safe use of FRUZAQLA during breast-feeding has not been established. It is not known whether FRUZAQLA or its metabolites are excreted in human milk. There are no animal data on the excretion of FRUZAQLA in animal milk. A risk to the breastfeeding newborns/infants cannot be excluded. Breastfeeding should be discontinued during treatment and for 2 weeks after the last dose; Fertility: There are no data on the effects of FRUZAQLA on human fertility. Results from animal studies indicate that FRUZAQLA may impair male and female fertility. SPECIAL WARNINGS AND PRECAUTIONS FOR USE Hypertension: Hypertension, including hypertensive crisis, has been reported in patients treated with FRUZAQLA. Pre-existing hypertension should be monitored and adequately controlled in accordance with standard medical practices before starting FRUZAQLA treatment. Hypertension should be medically managed with antihypertensive medicinal products and adjustment of the FRUZAQLA dose, if necessary. FRUZAQLA should be permanently discontinued for hypertension that cannot be controlled with antihypertensive therapy or in patients with hypertensive crisis. Hemorrhagic events: Hemorrhagic events have been reported in patients treated with FRUZAQLA, including gastrointestinal (GI) tract events. Serious and sometimes fatal bleeding events have been reported in patients after treatment with FRUZAQLA. Hematologic and coagulation profiles should be monitored in accordance with standard medical practices in patients at risk for bleeding, including those treated with anticoagulants or other concomitant medicinal products that increase the risk of bleeding. In the event of severe bleeding requiring immediate medical intervention, FRUZAQLA should be permanently discontinued. Gastrointestinal perforation: GI perforation events, including fatal events, have been reported in patients treated with FRUZAQLA. Symptoms of GI perforation should be periodically monitored during treatment with FRUZAQLA. FRUZAQLA should be permanently discontinued in patients developing GI perforation. Proteinuria: Proteinuria events have occurred in patients treated with FRUZAQLA. Proteinuria should be monitored before initiation and during treatment with FRUZAQLA in accordance with standard medical practices. If urine dipstick proteinuria ≥ 2 g / 24 hours is detected, dose interruptions, adjustments, or discontinuation may be necessary. FRUZAQLA should be permanently discontinued in patients developing nephrotic syndrome. Palmar-plantar erythrodysesthesia syndrome (PPES): PPES is the most frequently reported dermatological adverse reaction. If Grade ≥2 skin reactions are detected, dose interruptions, adjustments, or discontinuation may be necessary. Posterior reversible encephalopathy syndrome (PRES): PRES has been reported in 1 patient (0.1%) treated with FRUZAQLA in clinical studies. PRES is a rare neurologic disorder that can present with headache, seizure, lethargy, confusion, altered mental function, blindness, and other visual or neurological disturbances, with or without associated hypertension. A diagnosis of PRES requires confirmation by brain imaging, preferably magnetic resonance imaging (MRI). In patients developing PRES, discontinuation of FRUZAQLA, along with control of hypertension and supportive medical management of other symptoms, are recommended.Impaired wound healing: Impaired wound healing has been reported in 1 patient (0.1%) treated with FRUZAQLA in clinical studies. Patients are recommended to withhold FRUZAQLA for at least 2 weeks prior to surgery. FRUZAQLA should not be resumed for at least 2 weeks after surgery, as clinically indicated when there is evidence of adequate wound healing. Arterial and venous thromboembolic events: It is recommended to avoid starting treatment with FRUZAQLA in patients with a history of thromboembolic events (including deep vein thrombosis and pulmonary embolism) within the past 6 months or if they have a history of stroke and/or transient ischemic attack within the last 12 months. If arterial thrombosis is suspected, FRUZAQLA should be discontinued immediately. INTERACTIONS Effects of other medicinal products on the pharmacokinetics of FRUZAQLA CYP3A inducers Co-administration of FRUZAQLA with rifampicin (a strong CYP3A inducer) 600 mg once daily decreased FRUZAQLA AUCinf by 65% and decreased Cmax by 12%. The concomitant use of FRUZAQLA with strong and moderate CYP3A inducers should be avoided. CYP3A inhibitors Co-administration of FRUZAQLA with itraconazole (a strong CYP3A inhibitor) 200 mg twice daily did not result in clinically meaningful changes in the area under the concentration-time curve (AUC) and Cmax of FRUZAQLA. No dose adjustment of FRUZAQLA is needed during concomitant use with CYP3A inhibitors. Gastric acid lowering agents Co-administration of FRUZAQLA with rabeprazole (a proton pump inhibitor) 40 mg once daily did not result in clinically meaningful changes in the AUC of FRUZAQLA. No dose adjustment of FRUZAQLA is needed during concomitant use with gastric acid lowering agents. Effect of FRUZAQLA on the pharmacokinetics of other medicinal products Medicinal products that are substrates of P-glycoprotein (P-gp) Co-administration of a single dose of dabigatran etexilate 150 mg (a P-gp substrate) with a single dose of FRUZAQLA 5 mg decreased AUC of dabigatran by 9%. No dose adjustment is recommended for P-gp substrates during concomitant use with FRUZAQLA. Medicinal products that are substrates of breast cancer resistance protein (BCRP) Co-administration of a single 10 mg dose of rosuvastatin (a BCRP substrate) with a single 5 mg dose of FRUZAQLA decreased AUC of rosuvastatin by 19%. No dose adjustment is recommended for BCRP substrates during concomitant use with FRUZAQLA. UNDESIRABLE EFFECTS: The most commonly reported adverse reactions with FRUZAQLA are: Very common(frequency ≥1/10)Thrombocytopenia, hypothyroidism, anorexia, hypertension, dysphonia, diarrhoea, stomatitis, aspartate aminotransferase increased, total bilirubin increased, alanine aminotransferase increased, palmar-plantar erythrodysesthesia syndrome, musculoskeletal discomfort, arthralgia, proteinuria, asthenia, and fatigueCommon(≥1/100 to <1/10)Pneumonia, upper respiratory tract infection, bacterial infections, leukopenia, neutropenia, hypokalemia, epistaxis, throat pain, gastrointestinal hemorrhage, gastrointestinal perforation, pancreatic enzymes increased, oral pain, rash, and mucosal inflammation For US Prescribing Information: https://www.fruzaqla.com/sites/default/files/resources/fruzaqla-prescribing-information.pdf For Japan Prescribing Information: https://www.pmda.go.jp/PmdaSearch/iyakuDetail/ResultDataSetPDF/400256_42910H0M1028_1_01 Forward-Looking Statements This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the US Private Securities Litigation Reform Act of 1995. These forward-looking statements reflect HUTCHMED’s current expectations regarding future events, including but not limited to, its expectations regarding the receipt of the milestone payment, the therapeutic potential of fruquintinib for the treatment of such patients with CRC and the further clinical development of fruquintinib in this and other indications. Forward-looking statements involve risks and uncertainties. Such risks and uncertainties include, among other things, assumptions regarding the sufficiency of clinical data to support approval of fruquintinib for the treatment of patients with CRC or other indications in other jurisdictions such as Japan, its potential to gain approvals from regulatory authorities, the safety profile of fruquintinib, HUTCHMED and/or Takeda’s ability to fund, implement and complete its further clinical development and commercialization plans for fruquintinib, the timing of these events, each party’s ability to satisfy the terms and conditions under the license agreement; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials or the regulatory pathway for fruquintinib; and Takeda’s ability to successfully develop and commercialize fruquintinib. In addition, as certain studies rely on the use of other drug products as combination therapeutics with fruquintinib, such risks and uncertainties include assumptions regarding the safety, efficacy, supply and continued regulatory approval of these therapeutics. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. For further discussion of these and other risks, see HUTCHMED’s filings with the US Securities and Exchange Commission, on AIM and on The Stock Exchange of Hong Kong Limited. HUTCHMED undertakes no obligation to update or revise the information contained in this press release, whether as a result of new information, future events or circumstances or otherwise. Medical Information This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development. CONTACTS Investor Enquiries+852 2121 8200 / ir@hutch-med.com Media Enquiries Ben Atwell / Alex Shaw, FTI Consulting+44 20 3727 1030 / +44 7771 913 902 (Mobile) / +44 7779 545 055 (Mobile) / HUTCHMED@fticonsulting.comZhou Yi, Brunswick+852 9783 6894 (Mobile) / HUTCHMED@brunswickgroup.com Nominated Advisor Atholl Tweedie / Freddy Crossley / Rupert Dearden, Panmure Liberum+44 (20) 7886 2500 __________________________ REFERENCES 1American Cancer Society. Cancer Facts & Figures 2024. Atlanta, American Cancer Society; 2024.2Bray F, et al. Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 Countries. CA Cancer J Clin. 2024;74(3):229-263. doi:10.3322/caac.21834.3Ferlay J, et al. Global Cancer Observatory: Cancer Today. Lyon, France: International Agency for Research on Cancer. Available from: https://gco.iarc.who.int/today, accessed 12 June 2024.4Bando H, et al. Therapeutic landscape and future direction of metastatic colorectal cancer. Nat Rev Gastroenterol Hepatol 2023; 20(5)306-322. doi:10.1038/s41575-022-00736-1.5D'Haene N, et al. Clinical application of targeted next-generation sequencing for colorectal cancer patients: a multicentric Belgian experience. Oncotarget. 2018;9(29):20761-20768. Published 2018 Apr 17. doi:10.18632/oncotarget.25099.6Venderbosch S, et al. Mismatch repair status and BRAF mutation status in metastatic colorectal cancer patients: A pooled analysis of the CAIRO, CAIRO2, COIN, and FOCUS Studies. Clinical Cancer Res. 2014; 20(20):5322–5330. doi:10.1158/1078-0432.ccr-14-0332.7Koopman M, et al. Deficient mismatch repair system in patients with sporadic advanced colorectal cancer. Br J Cancer. 2009;100(2), 266–273. doi:10.1038/sj.bjc.6604867.8Ahcene Djaballah S, et al. HER2 in Colorectal Cancer: The Long and Winding Road From Negative Predictive Factor to Positive Actionable Target. Am Soc Clin Oncol Educ Book. 2022;42:1-14. doi:10.1200/EDBK_351354.9Dasari NA, et al. Fruquintinib versus placebo in patients with refractory metastatic colorectal cancer (FRESCO-2): an international, multicentre, randomised, double-blind, Phase III study. Lancet. 2023;402(10395):41-53. doi:10.1016/S0140-6736(23)00772-9.10Li J, et al. Effect of Fruquintinib vs Placebo on Overall Survival in Patients With Previously Treated Metastatic Colorectal Cancer: The FRESCO Randomized Clinical Trial. JAMA. 2018;319(24):2486-2496. DOI:10.1001/jama.2018.7855.

The federal 340B program has become a thorn in the side of pharmaceutical companies, who point to the drug discount program’s sprawling growth amid what they contend is lack of oversight. After initially proposing changes to the way the company offers drugs under this program, Johnson & Johnson has backed down, averting further confrontation with federal officials and hospitals on the matter. For now. When the 340B program was established in 1992, the intent was to help underserved communities. Under the program, eligible hospitals and clinics serving low-income and uninsured patients may purchase outpatient prescription medications at discounts of up to 50%. In August, J&J proposed a major change. Starting on Oct. 15, entities covered under 340B would have to pay full price, then make a claims submission for a rebate. This proposal would apply to two drugs, the plaque psoriasis medication Stelara and the blood thinner Xarelto. The Health Resources and Services Administration (HRSA), which administers the 340B program, told J&J the proposed changes are “inconsistent” with the statute and would need approval before they could take effect. In a Sept. 27 letter to J&J, HRSA Administrator Carole Johnson noted that the company had not requested such approval and proceeding with the rebate change would violate the 340B statute. The administrator added that implementations of the rebates would lead to termination of the company’s pharmaceutical pricing agreement and a referral to the Department Health and Human Services’ Office of the Inspector General. presented by Health Tech What’s Keeping Healthcare CIOs Up at Night: How to Improve Patient Satisfaction by Handling Calls More Efficiently Health systems have spent billions on portals while investments in modernizing the voice channel — the dominant preference of healthcare consumers — have taken a backseat. By Stephanie Baum In a letter of response, J&J maintained that the statute allows for rebates as a mechanism for drug manufacturers to offer the 340B price to covered entities. Furthermore, the company said its rebate proposal will target duplicate discounts, transactions in which a covered entity receives a 340B discount and then submits a claim for a Medicaid rebate — a violation of the statute. J&J says audits to uncover such duplicates have not solved the problem. “As HRSA knows, J&J’s efforts to identify and address program abuse through HRSA-approved audits of covered entities have been thwarted; nearly all of the entities for whom HRSA approved J&J audit requests have violated federal law by refusing to cooperate,” the company’s letter said. “Some have gone so far as to file suit against HRSA to invalidate the audit approvals. Plainly, audits alone are not a viable means for J&J to obtain information necessary to detect unlawful duplicate discounts and diversion as they occur.” Nevertheless, J&J said HRSA’s threat to terminate the company’s participation in the program would have the effect of cutting off millions of Medicare and Medicaid patients from necessary medicines. The company’s commitment to these patients leaves it “no choice but to forego implementation of the rebate model pending resolution of these issues.” J&J hints that litigation could be a next step. The company says it continues to believe its rebate model is “legally permissible” and “sorely needed” for drugmakers to comply with requirements of both the Inflation Reduction Act and the 340B statute. “J&J reserves all of its legal rights with respect to this matter,” the company said. presented by Health IT Accelerating Claim Processing: Strategies to Shorten the Life of a Claim These strategies and practices can significantly shorten the life cycle of claims, leading to quicker resolutions and improved financial outcomes. By Greenway Health In other regulatory news, we have FDA decisions for drugs (mostly approvals), some clinical holds, and one product withdrawal. Here’s a recap of recent regulatory developments: Regulatory Decisions —Exact Sciences’ Cologuard Plus now has FDA approval. The product is a next-generation version of Cologuard, the company’s non-invasive colorectal cancer test. Cologuard Plus offers greater sensitivity for colorectal cancer and advanced precancerous lesions. Like Cologuard, Cologuard Plus is approved for use in adults age 45 and older who are at average risk of colorectal cancer. Exact Sciences said it expects to launch this new test in 2025. —Partners Fresenius Kabi and Formycon received FDA approval for Otulfi, a biosimilar referencing the Johnson & Johnson biologic drug Stelara. The regulatory decision for Otulfi covers all of the approved uses of Stelara, which include Crohn’s disease, ulcerative colitis, moderate-to-severe plaque psoriasis, and active psoriatic arthritis. Separately, the European Union approved Otulfi in late September. Otulfi will jostle for market share with Pyzchiva from Samsung Bioepis and Sandoz, and Wezlana from Amgen, two Stelara biosimilars that have received FDA approvals in the past year. —Eli Lilly drug Retevmo is now FDA approved for treating adults and pediatric patients age 2 and older who have advanced cases of medullary thyroid cancer carrying a RET mutation. The drug has been available in this indication under accelerated approval. The latest regulatory decision converts Retevmo’s status to traditional approval. —Dupixent, a blockbuster immunology drug from Sanofi and Regeneron Pharmaceuticals, expanded its approved uses to include chronic obstructive pulmonary disease (COPD). Dupixent is the first FDA-approved biological treatment for COPD. Dupixent approved uses now span six dermatological and respiratory conditions. —FDA approval of Bristol Myers Squibb drug Cobenfy makes the drug the first novel schizophrenia drug in decades. The twice-daily capsule is designed to target a different receptor than currently available antipsychotic drugs, offering better efficacy and tolerability. Cobenfy comes from Karuna Therapeutics, which BMS acquired for $14 billion. —Eli Lilly drug Kisunla is now approved in Japan for the treatment of patients in the early stages of Alzheimer’s disease. Kisunla is part of a class of antibody drugs that work by reducing plaques of amyloid proteins that form in the brains of Alzheimer’s patients. Japan marks the second major market approval for Kisunla, which was approved by the FDA in July. —In other Japanese regulatory news, Takeda Pharmaceutical’s Fruzaqla won approval for the treatment of advanced or recurrent colorectal cancer. The Japanese pharma giant acquired the oral small molecule from Hutchmed in early 2023. Nearly a year ago, the FDA approved the drug for treating colorectal cancer. —Ipsen landed European Union approval for Kayfanda as a treatment for cholestatic pruritus (severe itching) associated with the rare liver disease Alagille syndrome. The approval was made under “exceptional circumstances” that permits marketing authorization when comprehensive efficacy and safety data are not available. The Ipsen drug won FDA approval for Alagille patients last year; in the U.S., the once-daily capsule is marketed as Bylvay. Ipsen added the drug to its pipeline via its $952 million acquisition of Albireo. —Ipsen’s rare liver disease portfolio also welcomed European Union approval of Iqirvo for treating primary biliary cholangitis (PBC), a chronic disorder that damages the liver’s bile ducts. The approval covers use of the once-daily pill in combination with ursodeoxycholic acid (UDCA) for patients whose disease does not adequately respond to that standard of care drug. Iqirvo may be used as a monotherapy for patients who cannot tolerate UDCA. European approval comes nearly four months after the FDA greenlit Iqirvo for PBC. —Zevra Therapeutics drug Miplyffa became the first FDA-approved treatment for Niemann-Pick disease type C, a rare inherited lysosomal storage disorder that can become fatal by the time patients reach adolescence. The approval specifically covers the neurological effects of the disease. Zevra acquired the rights to Miplyffa from Orphazyme, which failed to win FDA approval for the small molecule in 2021. —Days after Miplyffa’s approval, the FDA approved levacetylleucine, brand name Aqneursa, making the drug from privately held IntraBio the second approved treatment for Niemann-Pick disease type C. Similar to Miplyffa, the regulatory decision for the IntraBio drug covers the treatment of neurological effects of the rare disease. —The European Union approved Astellas drug zolbetuximab, brand name Vyloy, as a treatment for advanced gastric and gastroesophageal junction cancer. The approval covers use of the drug in combination with chemotherapy. The FDA turned down Astellas’s submission for the drug early this year, citing manufacturing issues. Astellas resubmitted its application, which now has a Nov. 9 target date for a regulatory decision. —FluMist, an intranasally dosed influenza vaccine that’s been available in the U.S. for two decades, is now FDA-approved for self- or caregiver administration. The regulatory decision makes the AstraZeneca product the first influenza vaccine that does not need to be given by a healthcare provider. For those choosing this option, the vaccine will be available through a third-party pharmacy following a screening and eligibility assessment completed when patients order the vaccine. —The FDA rejected Vanda Pharmaceuticals’ tradipitant as a treatment of gastroparesis. This delayed gastric emptying is associated with diabetes but can also develop in those who do not have diabetes. According to Vanda, the FDA disregarded clinical evidence for the drug and asked the company to conduct additional clinical testing. The company said it still plans to seek marketing authorization for this drug. A separate new drug application is also planned for tradipitant later this year for preventing vomiting from motion sickness. —An experimental Applied Therapeutics drug for the rare genetic metabolic disease classic galactosemia will not be discussed by an FDA advisory committee. After completing a late-cycle review meeting, the agency told the company a meeting was no longer needed for the drug, govorestat. The Nov. 28 target date for a regulatory decision still stands. —The FDA authorized the first over-the-counter hearing aid software. Called Hearing Aid Feature, this Apple software is compatible with the company’s AirPods Pro headphones. The software was reviewed through the regulator’s De Novo premarket review pathway. FDA authorization is based on a clinical trial enrolling 118 patients with mild-to-moderate hearing loss. Results showed those who self-fit the software and device achieved similar benefit as those who received a professional fitting. —Eli Lilly won FDA approval for lebrikizumab, brand name Ebglyss, a new treatment for atopic dermatitis. It will compete with biologic drugs Dupixent from Sanofi and Regeneron Pharmaceuticals, and Adbry from LEO Pharma. Compared to those every-other-week injectable drugs, Lilly’s new product offers patients less burdensome once-monthly maintenance dosing. —Roche landed FDA approvals for subcutaneously injectable drugs that were originally developed as intravenous infusions. Tecentriq Hybreza is the injectable version of the cancer immunotherapy Tecentriq; Ocrevus Zunovo is the injectable version of the multiple sclerosis drug Ocrevus. Both drugs employ drug delivery technology from Halozyme that enables biologic drugs to be administered as injections. —Travere Therapeutics drug Filspari converted its accelerated approval into a full FDA approval for the treatment of the rare kidney disease immunoglobulin A nephropathy. The regulatory decision came nearly a year after Travere reported the drug narrowly failed its Phase 3 confirmatory study. The approval is based on longer-term data showing the once-daily pill significantly slowed the kidney function decline. One Clinical Hold Placed, Two Lifted —Following the report of patient deaths, the FDA formally placed a clinical hold on zetomipzomib, a Kezar Life Sciences drug in development for treating lupus nephritis. Kezar had voluntarily stopped dosing and enrollment in the study after emerging safety data showed four Grade 5 (fatal) serious adverse events over the course of the study so far. The company said that three of those fatalities showed a common pattern of symptoms and proximity to dosing; additional non-fatal complications also showed a proximity to dosing. —The FDA lifted a clinical hold on Biomea Fusion’s Phase 1/2 clinical trial testing BMF-219 in type 1 and type 2 diabetes. Redwood City, California-based Biomea said review of the clinical data to date found that concerning safety signals observed in the Phase 2a escalation study did not translate to the larger Phase 2b expansion study. BMF-219 is a small molecule that forms a covalent bond and inhibits menin, a protein thought to suppress the pancreatic beta cells that produce and secrete insulin. —The FDA removed the partial clinical hold placed on an Avidity Biosciences’ delpacibart etedesiran, or del-disiran, an RNA therapy in development for the rare muscular disorder myotonic dystrophy type 1. The partial hold was placed in 2022 following the report of a serious adverse event in a single patient. Del-disiran is currently in Phase 3 testing. A Rare Disease Drug Withdrawal —Pfizer is voluntarily withdrawing Oxbryta from the market and discontinuing all clinical tests of the drug, which won accelerated FDA approval for sickle cell disease in 2019. The pharma giant said its decision is based on the totality of data now indicating higher rates of complications and deaths in patients treated with the oral drug. The FDA issued an alert about the withdrawal and said it has been conducting its own safety review of postmarketing data. When the review concludes, the agency said it will communicate additional findings, if necessary. Oxbryta was the centerpiece of Pfizer’s $5.4 billion acquisition of Global Blood Therapeutics in 2022.