Regulatory
The EMA approved a kidney disease–related label expansion for the blockbuster GLP-1 drug after a study showed reduced risk of death by 20%.
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.
In the U.S., the chorus of opposition against the proposed buyout continues to grow and now includes the CEOs of Roche and Lilly, a broad coalition of unions and consumer groups and at least one senator.
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.
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.
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.
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