Elamipretide Hydrochloride

iStock, Natalya Kosarevich The missed PDUFA adds to a string of delays at the FDA in recent weeks, including at least two other missed target action dates. The FDA has missed another target decision date, this time for GSK , which is looking to expand its IL-5 blocker Nucala into chronic obstructive pulmonary disease. As per a December 2024 press release from GSK announcing the FDA’s acceptance of its application, the regulator should have released a verdict on Wednesday, May 7. But several media outlets on Thursday reported that an announcement has yet to be made. In a statement to Fierce Pharma , a GSK spokesperson confirmed that the company has not received a decision from the FDA, but noted that it does “not comment on ongoing discussions with regulatory authorities.” “The FDA is actively reviewing our submission for Nucala in COPD [chronic obstructive pulmonary disease] and we are working closely with them to help bring this important treatment option to patients as quickly as possible,” the spokesperson told Fierce. “Based on our latest discussions with the FDA, we continue to expect approval.” BioSpace has reached out to GSK for independent confirmation of the delay and will update this article accordingly. The FDA in recent weeks has started to show lapses in its regulatory function and delays with its review process. Last month, for instance, the agency failed to provide Novavax with a verdict for its updated COVID-19 shot by the target date of April 1. The vaccine remains in limbo , though the company stands by its application—in late April, Novavax insisted that the shot was “approvable” and revealed that the FDA has asked for a post-marketing commitment to collect additional data. The agency similarly failed to meet its target decision date for Stealth BioTherapeutics’ elamipretide, an investigational drug for the ultra-rare disease Barth Syndrome. According to the biotech’s news release, the FDA should have released a verdict on April 29—but the regulator informed the company on the day itself that it would not meet that target action date. Earlier this week, the FDA put out a Federal Register notice announcing an upcoming meeting for its vaccine advisers. The posting, however, fell short of the customary 15-day advance notice for such meetings. The panel discussion is scheduled for May 22, while the Register notice was posted on May 8. The FDA itself conceded that it came up short: “FDA regrets that it was unable to publish this notice 15 days prior to the [meeting] due to technical issues.” Still, the agency is pushing through with the meeting, citing the “exceptional circumstances” that warrant the expert discussion. Since Robert F. Kennedy Jr. assumed the role of Secretary of Health and Human Services in February, the FDA has lost some 3,500 employees to layoffs, though the number is likely higher owing to voluntary exits.

This isn’t the first time—or even the second—that the FDA has fumbled its review timelines this year. In the wake of mass restructuring efforts across the Department of Health and Human Services (HHS), the FDA has missed yet another approval decision target date.GSK had expected to secure an FDA nod for its IL-5 antibody Nucala in chronic obstructive pulmonary disease (COPD) on Wednesday, but the May 7 deadline has come and gone without a decision from the U.S. regulator.“Based on our latest discussions with the FDA, we continue to expect approval,” a GSK spokesperson said in an email.“The FDA is actively reviewing our submission for Nucala in COPD and we are working closely with them to help bring this important treatment option to patients as quickly as possible,” the spokesperson said, caveating that the company does “not comment on ongoing discussions with regulatory authorities.”GSK had been hoping to make the COPD nod Nucala’s fifth from the FDA. Should Nucala win FDA approval in COPD down the line, the biologic will go toe-to-toe with Sanofi and Regeneron’s immunology stalwart Dupixent, which picked up a green light in the indication in September. This isn’t the first time—or even the second—that the FDA has missed a decision target date this year.Early last month, the agency missed its April 1 target date to weigh in on a full approval for Novavax’s protein-based COVID-19 vaccine. Novavax confirmed in an email that the FDA had missed its deadline, noting that it had “responded to all of the FDA’s information requests” and felt that its shot was ready for approval.More recently, the FDA once again delayed its decision to approve Stealth BioTherapeutics’ candidate elamipretide in the ultrarare genetic disease Barth syndrome. In late April, the FDA told Stealth it wouldn’t meet its April 30 deadline and did not set a new decision target date."The FDA cannot comment on any pending application to preserve the integrity of the review process," a spokesperson for the HHS told Fierce Biotech at the time."Any delays in drug approvals are the result of the FDA’s rigorous review process, which is designed to ensure that all medications are safe and effective for the American people," the government spokesperson added. "These delays are not connected to the recent reorganization."Under the Prescription Drug User Fee Act of 1992, the FDA commits to timelines of 10 or six months for standard or priority drug reviews, respectively. Companies seeking approval from the agency submit fees under the program, which in turn make up a significant portion of the FDA's overall budget. The user fee collection program both provides the FDA with the resources needed for its work and helps the industry hold the agency accountable to providing prompt and efficient reviews. While it’s hard to say for certain exactly what’s happening behind the FDA’s doors right now, the recent gutting of thousands of jobs across the HHS under Secretary Robert F. Kennedy Jr.—including 3,500 full-time FDA employees—has raised major concerns about the regulator’s ability to carry out key functions like inspections and timely reviews. Last month, a “former director” of the FDA’s Center for Biologics Evaluation and Research (CBER)—who resigned in March—told BMO Capital Markets that the agency is now at serious risk of fumbling its drug review timelines over the next year or two.BMO did not explicitly identify the expert it interviewed, though all signs point to it being Peter Marks, M.D., Ph.D., who exited the CBER in late March after allegedly butting heads with RFK Jr. around vaccine policy.The former director stressed that FDA staffers are still committed to conducting timely reviews. Nevertheless, major changes have transpired at the agency, and the developments that have been disclosed publicly are just the “tip of the iceberg,” the expert told BMO.