FDA
Analysts called the approval a much-needed win for Novo Nordisk, but warned that the company could struggle to grow sales once rival drugs come to market.
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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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The cancers were diagnosed 19 to 92 months after Skysona treatment.
After extending its review period to evaluate additional submissions, the FDA ultimately denied Applied Therapeutics’ govorestat for galactosemia, citing “deficiencies” with the application. The biotech plans to meet with the regulator to discuss the best way forward for the drug.
Despite hotly debated biomarkers and failed or delayed confirmatory trials, the accelerated approval program has a track record of propelling R&D for some of medicine’s most challenging illnesses.
Currently, Eli Lilly’s GLP-1 drugs Mounjaro and Zepbound (tirzepatide) are not on the FDA’s shortage list but compounded pharmacies are still making them. That’s unprecedented.
With two decisions originally scheduled for this week already announced, including BridgeBio’s approval in ATTR-CM, the regulator has just one PDUFA on its plate this holiday week.
RFK Jr. as HHS head is perhaps President-elect Donald Trump’s most controversial Cabinet pick now that Matt Gaetz has withdrawn as nominee for Attorney General. With Dr. Oz tapped to lead CMS and maybe Marty Makary at the FDA, it’s going to be quite the show.
The pharma is seeking full approval for its anticoagulation reversal drug Andexxa, which the FDA granted accelerated approval in 2018 for patients who had been treated with apixaban or rivaroxaban.
In this deep dive BioSpace explores the opportunities and challenges presented by the FDA’s accelerated approval program.
The FDA has followed in the footsteps of its European counterparts and granted accelerated approval to PTC Therapeutics’ gene therapy Kebilidi for AADC deficiency. It is the first approved gene therapy to be delivered directly to the brain.
Following patient deaths in a lupus trial that led to the termination of that program, Kezar’s autoimmune candidate zetomipzomib faces a partial clinical hold barring four trial participants from continuing treatment in the open-label portion of the trial, though the trial itself will continue as planned.