Mirvetuximab soravtansine

Reports Third-Quarter Diluted EPS of $0.88 on a GAAP Basis, a Decrease of 12.0 Percent; Adjusted Diluted EPS of $3.00, an Increase of 1.7 Percent; These Results Include an Unfavorable Impact of $0.04 Per Share Related to Acquired IPR&D and Milestones Expense Delivers Third-Quarter Net Revenues of $14.460 Billion, an Increase of 3.8 Percent on a Reported Basis or 4.9 Percent on an Operational Basis Third-Quarter Global Net Revenues from the Immunology Portfolio Were $7.046 Billion, an Increase of 3.9 Percent on a Reported Basis, or 4.8 Percent on an Operational Basis; Global Humira Net Revenues Were $2.227 Billion; Global Skyrizi Net Revenues Were $3.205 Billion; Global Rinvoq Net Revenues Were $1.614 Billion Third-Quarter Global Net Revenues from the Oncology Portfolio Were $1.687 Billion, an Increase of 11.6 Percent on a Reported Basis, or 13.0 Percent on an Operational Basis; Global Imbruvica Net Revenues Were $828 Million; Global Venclexta Net Revenues Were $677 Million Third-Quarter Global Net Revenues from the Neuroscience Portfolio Were $2.363 Billion, an Increase of 15.6 Percent on a Reported Basis, or 16.0 Percent on an Operational Basis; Global Botox Therapeutic Net Revenues Were $848 Million; Global Vraylar Net Revenues Were $875 Million; Combined Global Ubrelvy and Qulipta Net Revenues Were $445 Million Third-Quarter Global Net Revenues from the Aesthetics Portfolio Were $1.239 Billion, a Decrease of 0.1 Percent on a Reported Basis, or an Increase of 1.8 Percent on an Operational Basis; Global Botox Cosmetic Net Revenues Were $671 Million; Global Juvederm Net Revenues Were $258 Million Successfully Completed Acquisition of Cerevel, Adding Pipeline of Highly Complementary Assets to AbbVie's Existing Neuroscience Portfolio Raises 2024 Adjusted Diluted EPS Guidance Range from $10.67 - $10.87 to $10.90 - $10.94, which Includes an Unfavorable Impact of $0.64 Per Share Related to Acquired IPR&D and Milestones Expense Incurred Year-To-Date Through the Third Quarter 2024 Announces 2025 Dividend Increase of 5.8 Percent, Beginning with Dividend Payable in February 2025 NORTH CHICAGO, Ill., Oct. 30, 2024 /PRNewswire/ -- AbbVie (NYSE:ABBV) announced financial results for the third quarter ended September 30, 2024. "We delivered another quarter of strong commercial execution and significant pipeline progress," said Robert A. Michael, chief executive officer, AbbVie. "Based upon the momentum of AbbVie's business and our confidence in the long-term growth outlook, we are once again raising our full-year guidance and are increasing our quarterly dividend." Third -Quarter Results Worldwide net revenues were $14.460 billion, an increase of 3.8 percent on a reported basis, or 4.9 percent on an operational basis. Global net revenues from the immunology portfolio were $7.046 billion, an increase of 3.9 percent on a reported basis, or 4.8 percent on an operational basis. Global Humira net revenues of $2.227 billion decreased 37.2 percent on a reported basis, or 36.5 percent on an operational basis. U.S. Humira net revenues were $1.765 billion, a decrease of 41.6 percent. Internationally, Humira net revenues were $462 million, a decrease of 12.4 percent on a reported basis, or 7.8 percent on an operational basis. Global Skyrizi net revenues were $3.205 billion, an increase of 50.8 percent on a reported basis, or 51.5 percent on an operational basis. Global Rinvoq net revenues were $1.614 billion, an increase of 45.3 percent on a reported basis, or 47.4 percent on an operational basis. Global net revenues from the oncology portfolio were $1.687 billion, an increase of 11.6 percent on a reported basis, or 13.0 percent on an operational basis. Global Imbruvica net revenues were $828 million, a decrease of 8.8 percent, with U.S. net revenues of $618 million and international profit sharing of $210 million. Global Venclexta net revenues were $677 million, an increase of 14.8 percent on a reported basis, or 18.2 percent on an operational basis. Global Elahere net revenues were $139 million. Global net revenues from the neuroscience portfolio were $2.363 billion, an increase of 15.6 percent on a reported basis, or 16.0 percent on an operational basis. Global Botox Therapeutic net revenues were $848 million, an increase of 13.4 percent on a reported basis, or 14.4 percent on an operational basis. Global Vraylar net revenues were $875 million, an increase of 16.6 percent. Global Ubrelvy net revenues were $269 million, an increase of 15.3 percent. Global Qulipta net revenues were $176 million, an increase of 33.6 percent. Global net revenues from the aesthetics portfolio were $1.239 billion, a decrease of 0.1 percent on a reported basis, or an increase of 1.8 percent on an operational basis. Global Botox Cosmetic net revenues were $671 million, an increase of 8.2 percent on a reported basis, or 9.9 percent on an operational basis. Global Juvederm net revenues were $258 million, a decrease of 19.7 percent on a reported basis, or 16.9 percent on an operational basis. On a GAAP basis, the gross margin ratio in the third quarter was 70.9 percent. The adjusted gross margin ratio was 84.4 percent. On a GAAP basis, selling, general and administrative (SG&A) expense was 29.1 percent of net revenues. The adjusted SG&A expense was 23.0 percent of net revenues. On a GAAP basis, research and development (R&D) expense was 14.7 percent of net revenues. The adjusted R&D expense was 14.2 percent of net revenues. Acquired IPR&D and milestones expense was 0.6 percent of net revenues. On a GAAP basis, the operating margin in the third quarter was 26.5 percent. The adjusted operating margin was 46.7 percent. Net interest expense was $591 million. On a GAAP basis, the tax rate in the quarter was 25.0 percent. The adjusted tax rate was 16.2 percent. Diluted EPS in the third quarter was $0.88 on a GAAP basis. Adjusted diluted EPS, excluding specified items, was $3.00. These results include an unfavorable impact of $0.04 per share related to acquired IPR&D and milestones expense. Note: "Operational" comparisons are presented at constant currency rates that reflect comparative local currency net revenues at the prior year's foreign exchange rates. Recent Events AbbVie announced that it completed its acquisition of Cerevel, adding a pipeline of highly complementary assets to AbbVie's existing neuroscience portfolio. Cerevel's pipeline includes emraclidine, a potential best-in-class, next-generation antipsychotic, that is being studied for the treatment of schizophrenia; tavapadon, a first-in-class dopamine D1/D5 selective partial agonist for the management of Parkinson's disease (PD); as well as CVL-354, a potential best-in-class kappa opioid receptor (KOR) antagonist being studied for the treatment of major depressive disorder (MDD). Cerevel is a strong strategic fit for AbbVie and has potential to meaningfully impact revenue into the next decade. AbbVie announced positive topline results from its pivotal Phase 3 TEMPO-1 trial evaluating tavapadon as a fixed-dose monotherapy treatment in early PD. In the study, tavapadon met the primary endpoint, demonstrating a statistically significant improvement from baseline in the Movement Disorder Society - Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts II and III combined score at week 26. Tavapadon also met the key secondary endpoint, demonstrating statistically significant improvement from baseline in the MDS-UPDRS Part II score. Full results from the TEMPO-1 study will be submitted for presentation at future medical meetings and used to support regulatory submissions of tavapadon as a treatment for PD. Topline results from TEMPO-2, the Phase 3 flexible-dose monotherapy trial for tavapadon, are expected by the end of 2024. AbbVie announced the U.S. Food and Drug Administration (FDA) approved Vyalev (foscarbidopa and foslevodopa) as the first subcutaneous 24-hour infusion of levodopa-based therapy for the treatment of motor fluctuations in adults with advanced PD. The approval was supported by results from a pivotal Phase 3 head-to-head, randomized and controlled clinical trial that demonstrated a statistically significant improvement in "on" time without troublesome dyskinesia and decreased "off" time, compared to oral immediate-release carbidopa/levodopa (CD/LD). AbbVie and Aliada Therapeutics announced a definitive agreement under which AbbVie will acquire Aliada, a biotechnology company advancing therapies using a novel blood-brain barrier (BBB)-crossing technology to address challenging central nervous system (CNS) diseases. Aliada's lead investigational asset utilizing this delivery technology, ALIA-1758, is an anti-pyroglutamate amyloid beta (3pE-Aβ) antibody in development for the treatment of Alzheimer's disease (AD). The acquisition also allows AbbVie to utilize Aliada's novel BBB-crossing technology to enhance discovery and development efforts across neuroscience. AbbVie and Gedeon Richter announced a new discovery, co-development and license agreement to advance novel targets for the potential treatment of neuropsychiatric conditions. This collaboration expands upon the success of nearly two decades of partnership on CNS projects. AbbVie announced the European Commission (EC) approved Skyrizi (risankizumab) for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to conventional or biologic therapy. The approval was supported by data from two pivotal Phase 3 trials in which Skyrizi achieved the primary endpoint of clinical remission as well as key secondary endpoints. This marketing authorization for Skyrizi marks its fourth approved indication in the European Union (EU). Skyrizi is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally. At the European Academy of Dermatology and Venerology (EADV) Congress 2024, AbbVie shared more than 30 presentations that showcased the depth and strength of AbbVie's dermatology portfolio. Presentations highlighted data for Rinvoq (upadacitinib), Skyrizi and lutikizumab across a multitude of dermatological conditions. AbbVie announced that the EC granted conditional marketing authorization for Tepkinly (epcoritamab) as a monotherapy for the treatment of adult patients with relapsed or refractory (r/r) follicular lymphoma (FL) after two or more lines of prior therapy. Tepkinly is the first subcutaneous bispecific antibody conditionally approved as a monotherapy in the EU to treat both r/r FL and r/r diffuse large B-cell lymphoma (DLBCL), after two or more lines of prior therapy. The EC approval is supported by data from the Phase 1/2 EPCORE NHL-1 clinical trial, which evaluated the safety and efficacy of Tepkinly in adult patients with r/r FL. Tepkinly is being co-developed by AbbVie and Genmab. AbbVie announced the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending the marketing authorization of Elahere (mirvetuximab soravtansine) for the treatment of adult patients with folate receptor alpha (FRα)-positive, platinum-resistant and high-grade serous epithelial ovarian, fallopian tube or primary peritoneal cancer who have received one to three prior treatment regimens. The CHMP's opinion is supported by results of the Phase 3 MIRASOL clinical trial and the EC decision on this indication for Elahere is anticipated later this year. AbbVie announced submission of a Biologics License Application (BLA) to the FDA for accelerated approval (AA) of Teliso-V (telisotuzumab vedotin) in adult patients with previously treated, locally advanced or metastatic epidermal growth factor receptor (EGFR) wild type, nonsquamous non-small cell lung cancer (NSCLC) with c-Met protein overexpression. The BLA is supported by data from the Phase 2 LUMINOSITY clinical trial and review of the BLA will be conducted under FDA's Oncology Center of Excellence (OCE) Real-Time Oncology Review (RTOR) program. There are currently no approved anti-cancer therapies specifically for c-Met overexpressing NSCLC and if approved, Teliso-V would be the first-in-class therapy for this patient population. At the European Society for Medical Oncology (ESMO) Congress 2024, AbbVie showcased new data from its innovative antibody-drug conjugate (ADC) platform in tumor types with high unmet needs. Highlights included full data from the primary analysis of the positive, single-arm Phase 2 PICCOLO trial, evaluating Elahere for high FRα expressing platinum-sensitive ovarian cancer (PSOC); patient reported outcomes from the Phase 2 LUMINOSITY trial, evaluating Teliso-V in advanced NSCLC; as well as new safety and efficacy data in pre-treated patients with advanced NSCLC and gastroesophageal (GEA) cancer, from a Phase 1 study of ABBV-400 (telisotuzumab adizutecan). Allergan Aesthetics announced the FDA approved Botox Cosmetic (onabotulinumtoxinA) for temporary improvement in the appearance of moderate to severe vertical bands connecting the jaw and neck (platysma bands) in adults. Botox Cosmetic is the first product with four aesthetic indication areas: forehead lines, frown lines, crow's feet lines, and now platysma bands, making it the first product of its kind to go beyond the face. Allergan Aesthetics announced the launch of Botox Cosmetic for the treatment of masseter muscle prominence (MMP) in China. The approval is supported by Botox Cosmetic's well-established safety profile as well as clinical trial data that demonstrated Botox Cosmetic is effective in reducing the prominence of the masseter muscle. Botox Cosmetic is the first neurotoxin approved in China for MMP, the largest global MMP market. Allergan Aesthetics intends to develop Botox Cosmetic treatment for MMP in additional global markets and expand the use of Botox Cosmetic in the lower face. At the American Society for Dermal Surgery (ASDS), Allergan Aesthetics presented a total of 12 abstracts that showcased its commitment to patient outcomes and detailed insights and understanding of key concerns across differentiated patient segments. Highlights included four Best of Cosmetic Abstracts as well as a panel discussion on the impact of social media on patient experience and expectations when considering aesthetic treatment. Full-Year 2024 Outlook AbbVie is raising its adjusted diluted EPS guidance for the full year 2024 from $10.67 - $10.87 to $10.90 - $10.94, which includes an unfavorable impact of $0.64 per share related to acquired IPR&D and milestones expense incurred year-to-date through the third quarter 2024. The company's 2024 adjusted diluted EPS guidance excludes any impact from acquired IPR&D and milestones that may be incurred beyond the third quarter of 2024, as both cannot be reliably forecasted. Any potential IPR&D and milestones expense related to the recently announced acquisition of Aliada Therapeutics is also excluded from AbbVie's 2024 adjusted diluted EPS guidance, as the transaction is expected to close in the fourth quarter of 2024. Company Declares Dividend Increase of 5.8 Percent AbbVie is announcing today that its board of directors declared an increase in the company's quarterly cash dividend from $1.55 per share to $1.64 per share beginning with the dividend payable on February 14, 2025 to shareholders of record as of January 15, 2025. This reflects an increase of approximately 5.8 percent, continuing AbbVie's strong commitment to returning cash to shareholders through a growing dividend. Since the company's inception in 2013, AbbVie has increased its quarterly dividend by 310 percent. AbbVie is a member of the S&P Dividend Aristocrats Index, which tracks companies that have annually increased their dividend for at least 25 consecutive years. About AbbVie AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience and eye care - and products and services across our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at . Follow @abbvie on X (formerly Twitter), Facebook, Instagram, YouTube or LinkedIn. Conference Call AbbVie will host an investor conference call today at 8:00 a.m. Central Time to discuss our third-quarter performance. The call will be webcast through AbbVie's Investor Relations website at investors.abbvie.com. An archived edition of the call will be available after 11:00 a.m. Central Time. Non-GAAP Financial Results Financial results for 2024 and 2023 are presented on both a reported and a non-GAAP basis. Reported results were prepared in accordance with GAAP and include all revenue and expenses recognized during the period. Non-GAAP results adjust for certain non-cash items and for factors that are unusual or unpredictable, and exclude those costs, expenses, and other specified items presented in the reconciliation tables later in this release. AbbVie's management believes non-GAAP financial measures provide useful information to investors regarding AbbVie's results of operations and assist management, analysts, and investors in evaluating the performance of the business. Non-GAAP financial measures should be considered in addition to, and not as a substitute for, measures of financial performance prepared in accordance with GAAP. Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions and uses of future or conditional verbs, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those expressed or implied in the forward-looking statements. Such risks and uncertainties include, but are not limited to, challenges to intellectual property, competition from other products, difficulties inherent in the research and development process, adverse litigation or government action, and changes to laws and regulations applicable to our industry. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2023 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its Quarterly Reports on Form 10-Q and in other documents that AbbVie subsequently files with the Securities and Exchange Commission that update, supplement or supersede such information. AbbVie undertakes no obligation, and specifically declines, to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. SOURCE AbbVie WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

iStock/ Lynne Mitchell As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market. Last month, Pfizer’s sickle cell disease therapy Oxbryta , which won accelerated approval in 2019, was pulled from market due to risk of death and other complications, illustrating the risk of greenlighting therapeutics through this pathway. The FDA’s accelerated approval program has helped shepherd nearly 300 new drugs to the market, providing access to these medicines an average of 3.2 years earlier than with traditional approvals, according to the industry trade group PhRMA. Focusing on five drugs approved through this program, PhRMA’s report found that “millions of patients have gained faster access to clinical benefits, gaining additional life years, years of symptom control, or disease complication-free years over standard of care.” Having originated during the AIDS pandemic, many accelerated approvals have come in infectious disease, as well as in oncology, but the pathway has recently been leveraged by several high-pro and rare disease therapeutics. Over the past five years, the FDA has granted accelerated approval to novel therapies for Alzheimer’s disease, amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy and more. While about half of drugs approved through this pathway, including Eisai and Biogen’s Alzheimer’s drug Leqembi and AbbVie’s ovarian cancer drug Elahere , have succeeded in securing traditional approval, Pfizer’s Oxbryta is but one example where the therapy was ultimately unsuccessful . Others include Biogen and Eisai’s first Alzheimer’s drug, Aduhelm, which Biogen discontinued in January 2024, and Takeda’s Exkivity, which was withdrawn from the lung cancer market in October 2023 after failing to meet the primary endpoint in a confirmatory study. “The advantage of an accelerated pathway is that the FDA can potentially move drugs to help people with terrible conditions forward quickly,” Joel Perlmutter, a professor of neurology at Washington University School of Medicine in St. Louis, told BioSpace . “The downside is how they do that.” Accelerated approval is granted on the basis of a surrogate endpoint—such as a biomarker of the disease—that is reasonably likely to predict clinical benefit. Companies are then required to conduct a confirmatory trial while the drug hits the market. Gene therapies for rare diseases would seem to be ideal candidates for accelerated approval, given that biomarkers feature prominently in these trials, Emil Kakkis, CEO of rare disease drug developer Ultragenyx, told BioSpace . Yet the accelerated approval pathway has so far been utilized for very few rare disease therapies. Kakkis noted just two: Sarepta Therapeutics’ Elevidys , approved in June 2023 for Duchenne muscular dystrophy (DMD), and bluebird bio’s Skysona , greenlit in September 2022 for cerebral adrenoleukodystrophy. Kakkis indicated he would like to see the pace pick up. In a 2011 study , he and a colleague estimated that better access to the accelerated approval pathway could greatly reduce development costs in the rare disease space, enabling development of three times as many rare disease drugs even if investment stayed flat. Moreover, Kakkis said, prioritizing primary disease activity biomarkers that measure the cause of the disease rather than clinical endpoints for approval is “a step forward in the evolution of drug development and our understanding of disease.” “We should start defining disease as the cause of disease, not the outcomes of disease,” he said. “And if we do that, we’ll actually treat disease better. We’ll treat it before it hurts you.” Accelerated Approval Evolves Beyond Infectious Disease and Oncology The accelerated approval program was implemented in 1992 in response to the HIV/AIDS epidemic. During the first 10 years of the program, 65% of drugs to receive accelerated approval were for infectious diseases. The next two decades saw a multitude of cancer drugs hit the market through this pathway—between 2012 and 2021, 83% of accelerated approvals were in oncology. Now, a third era, accelerated approvals for rare and neurological diseases, appears to be dawning. In June 2021, Biogen and Eisai’s Aduhelm won accelerated approval as the first disease-modifying treatment for Alzheimer’s disease. This was followed by BioMarin’s Voxzogo for achondroplasia in November 2021, Biogen’s Qalsody for a rare form of ALS in April 2023 and Elevidys in June 2023. The approvals of Aduhelm and Elevidys, in particular, set off a firestorm of controversy as experts questioned the clinical benefit of these products. The subsequent market withdrawal of Aduhelm and the confirmatory trial failure of Elevidys added fuel to the fire and sparked questions about the value of the accelerated approval program. Then came Oxbryta. Joseph Ross, a professor of medicine and public health at Yale School of Medicine, expressed concern that other FDA divisions do not appear to be heeding the hard-won learnings of the Oncology Center of Excellence. “We are essentially being forced to relearn the lessons that the Oncology Group learned over the past 25 years,” he told BioSpace . These lessons include the need to avoid confirmatory trial delays and focus on clinical outcomes, he said. The FDA did not respond to BioSpace ’s requests for comment. Better Understanding Biomarkers The accelerated approval pathway primarily relies on biomarkers that may predict subsequent clinical benefit, Perlmutter said. “Therein lies the whole issue.” Investigational medicines are traditionally considered based on their clinical benefit and risk profile, Perlmutter explained. “Using a biomarker to predict clinical benefit for both the benefit and the risk is nearly impossible.” Current biomarkers “will give an idea of target engagement,” Perlmutter said. “It rarely tells you whether it’s safe to do so, and that almost certainly requires clinical observation.” This is where the confirmatory trials come in. But these studies are often delayed. In 2022, NPR found that 42% of currently outstanding confirmatory studies either took more than a year to begin following an accelerated approval, or had not yet started. “Those follow-up studies are rarely done in a timely fashion,” Perlmutter noted. In the end, he added, drugs with “potentially harmful effects” and “without adequate benefits” can remain on the market. Oxbrtya was on the market for nearly five years, while Gilead Sciences’ Zydelig was available to patients with lymphoma for more than seven years before Gilead withdrew the drug in 2022 after failing to complete follow-up studies. Ross noted that the FDA does not require surrogate markers to be validated by clinical evidence of an association between the marker and clinical outcomes. “It seems like a fool’s errand to continue to allow products to be approved on the basis of the same fallible surrogate markers over and over,” he said. “I think we need a much better sense of the surrogate markers that are being used as efficacy endpoints.” Perlmutter offered Aduhelm, which won accelerated approval based on its ability to remove amyloid beta from the brains of patients with Alzheimer’s, as one example of a questionable biomarker. “The problem is, at that time—and actually currently—nobody [has] proved that reduction of amyloid in the brain predicts clinical benefit,” said Perlmutter, who served on the advisory committee that rejected Aduhelm and resigned as a result of its approval. But Kakkis contends that biomarkers are more of an issue when it comes to complex diseases like Alzheimer’s and ALS. “Those situations are trickier because you can’t be sure that the biomarker is reflecting the source of disease.” By contrast, biomarkers are a reliable surrogate endpoint in the rare, genetic disease space where they represent the underlying cause of the disease, he said. “In all cases, that’s transformed into clinical benefit down the road. It has not failed.” Perlmutter agreed that genetic diseases where the correlation between the biomarker and the cause of disease can be proven are an appropriate use of the accelerated approval pathway. “I just think you have to prove it before you approve it,” he said. A Worthy Regulatory Avenue There is a perception that accelerated approval is a regulatory shortcut , but Kakkis takes issue with the idea that it is “a less important or less rigorous approval.” “The biomarker-based approval of HIV drugs was not a compromise,” he said. “It’s far superior to doing it any other way.” He explained that it would have taken too long to run trials with clinical endpoints like opportunistic infections, hospitalizations or death with three or four combination drugs, “and too many people would have to get sick and die.” The FDA has taken steps to dispel this shortcut notion and tighten up the program. Responding to delays in the completion of confirmatory trials, legislation passed in 2022 empowers the agency to require these studies be underway before granting accelerated approval. But the experts who spoke with BioSpace say more changes are needed. Concerning confirmatory trials, Ross said, “I think we need much firmer deadlines that are much shorter in nature to ensure that we have timely evidence that helps the patient and clinician community quickly understand whether the drug is truly safe and effective.” Meanwhile, Perlmutter expressed concern about the potential for bias given that 65% of the FDA’s budget for human drugs comes from companies applying for approval. This is particularly an issue for accelerated approval “where judgment is critical and any potential bias can influence judgment,” he said. “Time is money for Big Pharma. Sooner approval means quicker onset of sales.”