FDA
The biologics center director reportedly became personally involved after the team reviewing the rare blood disorder filing asked for an extension to the CNPV-accelerated timeline.
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Representatives of companies including AbbVie, Eli Lilly, Johnson & Johnson and Merck have voiced concerns about the FDA’s approach to pre-approval inspections.
With notable therapies from Biogen, Sarepta and MacroGenics failing to show efficacy in pivotal or confirmatory trials, experts question the use of biomarker evidence for approval while one former regulator insists that a “failed trial is not a failed drug.”
Coming up in the back half of December, the FDA will issue a verdict on Vanda Pharmaceuticals’ gastroparesis drug tradipitant, which it rejected last September, triggering a very public dispute with the company.
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In its rejection letter, the FDA flagged problems at a third-party fill-finish site owned by Novo Nordisk. Issues at this site have previously been investigated by the regulator.
The agency also pointed to the use of Tylenol and other acetaminophen products during pregnancy as being potentially linked to neurological and developmental defects in children, following a press conference Monday in which President Donald Trump did the same.
An advisory committee last year found that Zynquista’s benefit-risk profile in type 1 diabetes was unfavorable due to cases of diabetic ketoacidosis.
The company was expecting a decision from the FDA by Sept. 28 for its oral drug tolebrutinib, but an update to the drug’s application package convinced the agency to take more time to review.
For the last two years, Keytruda has reigned as the world’s top-selling drug—a distinction under threat with key patent protections expiring in 2028.
A decade-long journey has come to an end for Stealth BioTherapeutics and the Barth syndrome community with the first-ever treatment for this uncommon mitochondrial disease. CEO Reenie McCarthy called it a “pivotal victory” that “offers hope for expedited regulatory attention to other ultra-rare diseases.”
Investors had been “holding out hope” that there remained a regulatory path forward for RP1, but results of Replimune’s Type A meeting with the FDA do not appear to support this, according to BMO Capital Markets.
Listen now for a high-level discussion on overcoming pharmaceutical supply chain challenges through end-to-end integration.
CDER Director George Tidmarsh said on two separate events last week that he thought advisory committee meetings for specific drug applications were redundant, but then denied having said the agency would abandon them.
On the FDA’s docket for the back half of September is Merck’s proposed subcutaneous formulation of its blockbuster cancer drug Keytruda.