FDA
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.
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.
Career conservative and former congressman Dave Weldon will, if confirmed, act as director of the Centers of Disease Control and Prevention, where his anti-vaccine views will mesh with those of selected Department of Health and Human Services head Robert F. Kennedy Jr.
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.
Despite the death, the FDA has allowed Neurogene to forge ahead with the Phase I/II Rett syndrome trial, but using only the lower 1E15 vg dose of its investigational gene therapy NGN-401.
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.
The agreement will give Kura enough capital to support the development and launch of its menin inhibitor ziftomenib.
PRESS RELEASES