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.
FEATURED STORIES
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.
Subscribe to ClinicaSpace
Clinical trial results, research news, the latest in cancer and cell and gene therapy, in your inbox every Monday
THE LATEST
Arguably the FDA’s most anticipated decision this month is for a subcutaneous induction formulation of Biogen and Eisai’s Alzheimer’s drug Leqembi, which, according to Eisai, could “help reduce the burden on healthcare professionals and patients.”
After exiting the FDA less than two weeks ago for unclear reasons, Vinay Prasad is once again director of the Center for Biologics Evaluation and Research, HHS confirmed to several outlets Saturday.
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.
In an open letter, 22 experts who designed and ran Replimune’s Phase III IGNYTE trial answered the FDA’s issues, as outlined in the complete response letter for the melanoma candidate RP1.
According to reporting from multiple outlets, Richard Pazdur, head of the Oncology Center of Excellence at CDER, opposed the consensus opinion of CBER staff to approve the drug. Replimune’s stock has dropped precipitously since the rejection.
Sarepta and Capricor learned of key regulatory decisions from the media and investors, and Duchenne muscular dystrophy families have turned to the news for answers. Meanwhile, the FDA insists it remains committed to notifying companies of any regulatory action before sharing information with the media or public.
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.
As analysts parsed news of Vinay Prasad’s ouster, worries over drug approval delays, cell and gene therapy impacts and more were top of mind.
The FDA’s dramatic summer continues to unfold as news broke late Tuesday evening that Vinay Prasad will depart the agency, where he had been the head of the Center for Biologics Evaluation and Research for less than three months.
The swift FDA action removes an overhang from Sarepta and allows Elevidys to return to the market without another safety study, as had been feared, Jefferies analysts said Monday.