FDA
Robert F. Kennedy, Jr.—Trump’s pick for HHS secretary who endured confirmation hearings last week—has repeatedly criticized industry ties to the FDA, particularly financial links between the two, which could indicate trouble for the user-fee model.
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RFK Jr. as HHS head is perhaps President-elect Donald Trump’s most controversial Cabinet pick now that Matt Gaetz has withdrawn as nominee for Attorney General. With Dr. Oz tapped to lead CMS and maybe Marty Makary at the FDA, it’s going to be quite the show.
In this deep dive BioSpace explores the opportunities and challenges presented by the FDA’s accelerated approval program.
As therapies for rare and neurological diseases earn accelerated approval, experts laud the program’s intent while remaining concerned about confirmatory trials and clinical efficacy, especially as products greenlit under this pathway are pulled from the market.
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Regulators squeezed in two final approvals before the calendar change with the UK approval of Merck’s Winrevair and the FDA’s greenlight for an injectable formulation of BMS’s cancer blockbuster.
Other notable greenlights this year include Bristol Myers Squibb’s Cobenfy, the first novel therapeutic for schizophrenia in 35 years, and Madrigal Pharmaceuticals’ Rezdiffra, the first-ever treatment for MASH.
The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
Despite securing the industry’s first approval for familial chylomicronemia syndrome, BMO Capital Markets believes that Tryngolza’s regulatory triumph will not be a significant positive for Ionis. Instead, the firm is focusing on olezarsen’s readout in severe hypertriglyceridemia, a much larger market.
The approval concludes what has been a difficult regulatory path for Ryoncil, which suffered FDA rejections in 2020 and 2023.
The letters come amid the Outsourcing Facilities Association’s ongoing lawsuit against the FDA over the regulator’s decision to end the shortage for tirzepatide.
The FDA cited issues with a manufacturing facility as the reason for the rejection. J&J is currently “working closely” with the regulator to resolve these problems.
The FDA has put a stop to U.S. initiation of PepGen’s Phase II trial for its Duchene muscular dystrophy treatment. The company faced the same hurdle for an earlier neuromuscular candidiate in 2023.
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%.
By speeding lifesaving drugs’ way to market and focusing on the underlying causes of disease, the pathway has helped save many lives.