Approvals
Novartis confirms that nine patients treated with its spinal muscular atrophy gene therapy Zolgensma have developed tumors; the FDA orders GLP-1 drugmakers to add a warning to their labels about the risk of a rare eye condition that can lead to vision loss; and the agency is set to decide on no fewer than 11 therapies this month, including closely watched applications from Summit Therapeutics, Vertex and Capricor Therapeutics.
Bayer’s Sunharbo has secured the first FDA approval of its class, establishing a regulatory pathway for other factor XIa inhibitors, such as Bristol Myers Squibb and Johnson and Johnson’s milvexian.
Also lined up are target action dates for Sanofi’s Gaucher disease therapy, GSK’s lung cancer medicine and BridgeBio’s muscular dystrophy drug.
Hibsago’s approval—a functional cure for certain patients with a hepatitis B infection—further validates Ionis Pharmaceuticals’ antisense platform, which has already generated two other new drug approvals this year.
Data released by the FDA show that Revolution Medicine’s newly approved pancreatic cancer drug underperformed in a large subgroup of patients, but analysts say the biotech still has a clear winner.
The approval of Mirum Pharmaceuticals’ ALK2 inhibitor for fibrodysplasia ossificans progressiva on Friday adds a third option for patients who had long been without any treatments, joining Regeneron’s recently approved Pasatru and Ipsen’s Sohonos.
In the Phase 3 TEMPO program, AbbVie’s Juvmo elicited significant improvements in motor symptoms and prolonged periods with good symptom control.
The weekly insulin Onswik will now be available to U.S. patients after initial approvals in Europe, Mexico and Japan.
The market approval for Fayuvi, which was rejected in 2025 due to manufacturing problems, comes shortly after Ultragenyx got the FDA nod for its first gene therapy last month.
Scholar Rock’s spinal muscular atrophy drug, now to be called Isembyld, was originally rejected in September 2025, when the FDA cited compliance problems at a third-party manufacturer.
PRESS RELEASES