Regulatory
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.
Suggestions that the U.S. should emulate other countries on drug price controls or patents obscure how our present policies have allowed drug development to flourish.
In February 2024, the FDA put two Phase II studies of zelnecirnon under a clinical hold after a case of liver failure was deemed potentially related to the drug.
With Novo Holdings’ $16.5 billion buyout of Catalent being reviewed by regulators, what work the contract drug manufacturer may or may not be performing for Eli Lilly remains a point of contention.
The contract manufacturer fell short of Wall Street’s projections for its first quarter of fiscal 2025, which ended Sept. 30, 2024. The miss comes amid growing opposition to Novo Holdings’ proposed $16.5 billion acquisition of Catalent.
Despite the PDUFA date being extended by three months for Merus’ zenocutuzumab, Truist Securities analyst Asthika Goonewardene in a Tuesday note to investors said the delay is not a cause for concern with an approval expected.
The FDA is set to decide on four promising therapies in the next two weeks, including a CAR T for acute lymphoblastic leukemia.
While an FDA advisory committee saw signals of efficacy for Lexicon Pharmaceuticals’ sotagliflozin, the panel of external experts found that the company did not provide enough data to support its drug application.
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