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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Vinay Prasad, chief of the FDA’s Center for Biologics Evaluation and Research, is planning to publish a paper this month to outline his office’s thinking on accelerating gene editing reviews.
The clinical hold comes days after Intellia voluntarily paused enrollment and dosing in the same two studies.
Generic versions of biologic drugs could hit the market without human trials and allow the market forces to bring down costs, HHS leaders said while announcing new draft guidance.
Despite the rejection, analysts saw Regeneron’s use of an alternate filler for Eylea HD as a positive development, with BMO Capital Markets noting that this could signal the end of manufacturing troubles for the franchise.
While the FDA continues to put out guidance documents and approve drugs, some companies are already reporting delays in dealings with the agency, while insiders warn of falling morale and a negative perception from the rest of the biopharma world.
Executives from Eli Lilly, Merck and other companies foresee the FDA’s new onshoring proposal being anything from a bureaucratic waste of time to a transformative program that will eliminate inspection-related complete response letters.
Lynkuet is the first FDA-approved therapy that blocks both the neurokinin 1 and neurokinin 3 receptors to treat hot flashes.
In three draft guidance documents, the FDA has laid out pointers to help companies craft better drug development programs and drug applications.
The antibody-drug conjugate, withdrawn from the market by GSK in 2022 after failing a confirmatory study, was approved Thursday by the FDA for previously treated patients with multiple myeloma. This is despite a negative advisory committee vote in July.
The company’s technology, a modified herpes simplex virus used to deliver gene therapies, was given the FDA’s new designation based on its approved topical skin cream. What this will mean for Krystal’s still-in-development eye drop is unclear.