FDA
Following news of RSV lower respiratory tract infections in infants immunized with Moderna’s investigational RNA vaccines, FDA advisors said the trial investigators should continue the study, while keeping an eye out for further safety signals.
Ocaliva recently failed to secure the FDA’s traditional approval for primary biliary cholangitis due to safety concerns.
In a Type B meeting, the FDA signified that it will allow uniQure to use a natural history control, the composite Unified Huntington’s Disease Rating Scale, and neurofilament light chain levels to support the accelerated approval of its gene therapy AMT-130.
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.
The FDA’s year-end rush includes nine target action dates, mostly for rare disease and cancer therapies.
The program has recently become controversial as a number of high-profile advanced approvals were granted only for the drugs to fail a confirmatory trial later on. Now, the FDA has clearly laid out expectations.
President-elect Donald Trump and his incoming administration are unlikely to attempt a wholesale restructuring of U.S. healthcare and could promote M&A activity, but controversial picks like Robert F. Kennedy could impact vaccine sales, experts say.
The agency’s warning letter outlines Applied Therapeutics’ failure to provide adequate information regarding the clinical trial—including a dosing error for govorestat—claims that the company said it had already addressed.
Novartis, Gilead, Roche and Takeda commit to new partners in a spate of mid-sized collaborations this week. Meanwhile, Applied Therapeutics’ stock tanks 80% after govorestat is denied approval, Intra-Cellular Therapies seeks to expand Caplyta into major depressive disorder and the FDA investigates the safety of bluebird bio’s Skysona.
The cancers were diagnosed 19 to 92 months after Skysona treatment.
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