FDA
The FDA in a complete response letter to Replimune maintained its original objection to the single-arm trial the biotech used to support the application for RP1.
FEATURED STORIES
The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.
The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher.
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Wednesday, the FDA’s VRBPAC agreed that GSK’s respiratory syncytial virus vaccine candidate is safe and effective in older adults.
The FDA greenlit Reata Pharmaceuticals’ omaveloxolone (Skyclarys) as the first and only treatment for Friedreich’s ataxia, a rare, life-limiting neuromuscular disorder.
The hits kept coming for the sickle cell disease space last week as the FDA placed a full clinical hold on Fulcrum Therapeutics’ Investigational New Drug application FTX-6058.
The FDA approved Sanofi and Sweden-based Sobi’s efanesoctocog alfa, now marketed as Altuviiio, to treat bleeding in adults and children with hemophilia A.
There are currently no disease-modifying treatments for Friedreich’s ataxia. That could change when the FDA makes a decision about Reata Pharmaceuticals’ omaveloxolone on Feb. 28.
After overcoming a clinical hold, Astellas presented preliminary safety and efficacy data from the Phase I/II FORTIS trial of AT845 in late-onset Pompe disease at the 19th Annual WORLDSymposium 2023.
On Friday, the FDA approved Apellis Pharmaceuticals’ Syfovre (pegcetacoplan) as the first treatment for geographic atrophy (GA), a leading cause of blindness.
Takeda’s vedolizumab met its primary endpoint in the Phase III GRAPHITE study, significantly improving event-free survival in patients with acute graft-versus-host disease.
The FDA granted accelerated approval Friday to Travere Therapeutics’ sparsentan, now to be marketed as Filspari, to reduce the burden of proteinuria in adults with primary IgA nephropathy.
The FDA’s Oncologic Drugs Advisory Committee voted 8-5 that two single-arm trials would be “sufficient to characterize the benefits and risks” of Jemperli in locally advanced rectal cancer.