Elamipretide Hydrochloride

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.

The biotech’s candidate, called elamipretide, had a PDUFA date scheduled for April 29.\n An approval decision for Stealth BioTherapeutics’ investigational treatment for an ultra-rare genetic disease has been delayed—again—this time, with no new decision date given.The biotech’s candidate, called elamipretide, had a Prescription Drug User Fee Action (PDUFA) date scheduled for today. However, the FDA said it wouldn’t meet the new drug application (NDA) deadline and didn’t give a new action date, according to an April 29 release from Stealth.The FDA did not return Fierce Biotech’s request for more information about the change.This isn’t the first FDA delay related to elamipretide, an injection designed to treat Barth syndrome, a life-threatening pediatric mitochondrial disease thought to impact less than 300 individuals globally.Stealth submitted an NDA on Jan. 29 of last year and received a PDUFA date of Jan. 29, 2025, under the standard review process. Later, the FDA granted the therapy priority review, with an advisory committee voting 10-6 in support of approving the drug in October.However, the FDA requested a three-month delay in January to review additional data supplied by Stealth. The FDA said the data counted as a major amendment to the approval request, triggering the standard three-month extension to the decision timeline.Now, while the FDA hasn’t shared a new expected action date, Stealth says labeling discussions between the agency and Massachusetts biotech are currently underway. “Despite the delay, Stealth remains optimistic and continues to engage with the FDA in this active review,” a Stealth spokesperson told Fierce Biotech. “Most important to address is the urgent need of those requiring expanded and emergency access to elamipretide.”The FDA previously granted elamipretide orphan drug, fast track and rare pediatric designations for Barth syndrome. But in October, the agency took issue with several parts of Stealth’s trials, saying that certain data were difficult to interpret and citing a potential placebo effect in the late-stage trial.“No other trial designs that had been discussed… were ultimately acceptable to FDA, primarily due to powering concerns that are frankly insurmountable in a disease that\'s rare,” Stealth CEO Reenie McCarthy said during the nine-hour advisory hearing.While Stealth isn’t the first biotech to experience delays since the Trump administration rapidly restructured the FDA, this also isn’t the first time Stealth has asked the agency to approve elamipretide.    The original application for the injection was tied to a phase 3 study, called SPIBA-001, that trial met its main goal. However, the data were compared to an open-label portion of a phase 2/3 crossover study and natural history controls—a strategy the FDA did not like.Back in 2021, the FDA said it expressed skepticism that the Stealth data could establish the effectiveness of elamipretide and recommended that Stealth launch a new phase 3 trial.Despite this, Stealth submitted an NDA seeking approval, prompting the FDA to issue a refusal-to-file letter, stating that the application didn’t contain a single adequate and well-controlled trial that could establish efficacy.The FDA continued to recommend a new late-stage trial, but Stealth informed the agency of its intent to resubmit the NDA without conducting a new trial. The regulator said it could not prevent an applicant from resubmitting and that Stealth should fully address the agency’s prior concerns in the resubmission.