Approvals
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.
The approval opens up a “modest” market for AstraZeneca’s Etcamah, according to Leerink analysts, who nevertheless looked forward to an upcoming late-stage readout that could lead to a “significantly larger opportunity” for the drug.
Zanvastro, an antisense oligonucleotide therapy, represents the first independent neurology launch for Ionis Pharmaceuticals, which intends to leverage the foothold for more approvals in rare neurodegenerative diseases, including Angelman syndrome.
The approval of rusfertide—now to be branded Mimrylo—for polycythemia vera marks a key win for Takeda, which now has crossed the FDA finish line for two of three critical product launches slated for the coming year.
PRESS RELEASES