FDA
Aiming to protect patients, the FDA sent lawmakers a wish list of legislative proposals intended to clarify and expand its oversight of updates to approved drug production processes.
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The FDA has some big verdicts lined up in the second quarter, including one for a closely watched obesity drug that many anticipate will further intensify competition in weight loss.
Draft guidance, issued by the FDA last week, could remove ambiguity and uncertainty that may have so far limited uptake of new approach methodologies, experts told BioSpace, particularly emphasizing the agency’s recommendations around defining NAMs’ regulatory purpose.
The FDA’s Commissioner’s National Priority Voucher program, unveiled in June 2025, is “shrouded in secrecy,” Democratic representative Jake Auchincloss said last month, as regulatory and biopharma leaders try to decode the criteria for investigational or approved drugs to receive a voucher.
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With two earlier trials meeting their primary endpoints, Axsome claimed it has the data to support a filing for FDA approval in the second half of 2025.
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%.