Accelerated approval

Stealth BioTherapeutics originally submitted elamipretide for approval in January 2024, only to receive a rejection in May of this year. In addition to accepting the biotech’s resubmission, the FDA has promised to take action on the candidate by Sept. 26.

The small molecule drug, acquired by Jazz Pharmaceuticals in its $935 million Chimerix pick-up this spring, is intended for relapsed adult and pediatric patients with H3 K27M mutations.

The FDA greenlit multiple new drugs this month and issued some notable label expansions, including for Eli Lilly’s Kisunla. Meanwhile, the regulator turned away a cell therapy for Duchenne muscular dystrophy and a gene therapy for the rare disease Sanfilippo syndrome.

The Commissioner’s National Priority Vouchers aim to offer accelerated pathways to drugs that meet certain criteria, perhaps including a low price-tag. But the policy is vaguely defined and was announced without public input, going against the FDA’s own published practices, experts say.

A journey through the FDA’s newly released complete response letters gave glimpses into the journeys to market for Eli Lilly’s Alzheimer’s antibody Kisunla, Sarepta’s DMD gene therapy Vyondys 53 and Gilead’s HIV drug Sunlenca.

While it trails Johnson & Johnson’s Tecvayli, Regeneron still hopes Lynozyfic can differentiate in terms of dosing convenience and efficacy.

Industry watchers responded mostly positively to the commissioner’s new voucher program, but worries remain over staffing cuts at the agency.

The program will bring together experts from across the FDA for a team-based review, rather than having an application move across numerous offices within the agency before getting a yay or nay.

AbbVie’s Emrelis is the first non-small cell lung cancer therapy approved for patients with high c-Met expression levels who have received prior lines of treatment.

An OIG report zeroed in on what it said were three particularly problematic accelerated approvals: Biogen’s Aduhelm, Sarepta’s Exondys and Covis’ Makena.