Regulatory
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.
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.
Biogen, Eli Lilly, Merck and Novartis spent more than $20 billion to absorb biotechs with promising or approved drugs; the rare disease space notched approvals for therapies from Denali Therapeutics, Rocket Pharmaceuticals and Biogen; and Wave’s stock lost half its value after its RNA-based obesity candidate failed to impress investors.
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.
Former ACIP vice chair Robert Malone claimed that Andrew Nixon, spokesperson for the Department of Health and Human Services, “trashed” him with the media, adding that he resigned because “I do not like drama.”
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.
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