FDA
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.
Makary is a pancreatic surgeon at Johns Hopkins who became known for battling medical mistakes and in recent years has been an outspoken critic of COVID-19 policies.
While the full impact of the Supreme Court decision remains unknown, the new regulatory landscape could be a net positive for drug developers.
In this deep dive BioSpace explores the opportunities and challenges presented by the FDA’s accelerated approval program.
Trump’s HHS pick, Robert F. Kennedy Jr., is an anti-vaccine campaigner who has previously said that he plans to gut the FDA on allegations of corruption and reduce the NIH’s headcount.
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.
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