FDA
The FDA has greenlit Travere Therapeutics’ Filspari as the only available treatment for focal segmental glomerulosclerosis despite the drug’s loss to Sanofi’s Avapro in a Phase 3 study.
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The upcoming FDA decision for Replimune’s advanced melanoma drug could be a litmus test for the agency’s future regulatory decision-making, analysts say, with implications stretching well beyond one company.
While recent FDA guidance speaks to the agency’s support of innovative trial designs—including the use of external controls—the application of this flexibility appears to be inconsistent. One former regulator says the situation is more nuanced.
With CBER director Vinay Prasad set to depart the agency at the end of the month, a coalition of patient groups and biotech executives penned a letter imploring the Trump administration to “restore regulatory clarity” for rare disease therapies. Experts on a BioSpace panel last week also acknowledged the challenges faced by a more stringent FDA.
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If the Trump administration’s proposal passes, the FDA’s budget will be more than $200 million bigger in 2027, with plans to launch new programs that expedite drug development, boost national security and promote “radical transparency.”
As Novo Nordisk cuts 400 jobs at the troubled site, Scholar Rock has seen enough progress that it included the facility in a resubmission for FDA approval.
The FDA approved Eli Lilly’s orforglipron—to be known as Foundayo—on Wednesday, officially igniting what analysts believe will be a fierce rivalry with Novo Nordisk’s oral Wegovy.
The FDA in January asked Amgen to pull Tavneos from the market, citing liver toxicity issues that affected the drug’s overall risk-benefit profile. The pharma refused.
The FDA rejected the high-dose regimen of Spinraza in September last year due to manufacturing concerns.
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.
While the accelerated approval unlocks only a small market opportunity for Rocket Pharmaceuticals, it will give the biotech a chance to prepare for future product launches, according to Jefferies.
Avlayah is the first Hunter syndrome therapy approved to address the condition’s neurologic complications, according to Tracy Beth Høeg, acting director of the Center for Drug Evaluation and Research.
While ersodetug missed the Phase 3 primary endpoint of a reduction in hypoglycemia events, Rezolute argued that this goal was confounded in part by behavioral factors. The FDA acknowledged the validity of this argument.
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.