The FDA has granted a priority review to the sNDA for Amvuttra, with a decision expected by March 23, 2025. If approved, Amvuttra will be the first therapeutic in the U.S. to treat both the polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis, potentially expanding the eligible patient population and boosting sales.
ALNY is also advancing its investigational RNAi therapeutic, mivelsiran, for Alzheimer’s disease and cerebral amyloid angiopathy. Interim data from part B of the phase I study in Alzheimer’s disease is expected in the second half of 2025, with a phase II study initiation planned for the same period.
In collaboration with Roche, ALNY is developing zilebesiran for hypertension, with phase II KARDIA-3 study data anticipated in the second half of 2025. A phase III cardiovascular outcomes study on zilebesiran is also planned to commence in the second half of 2025.
Alnylam has regained full global development and commercialization rights to mivelsiran across all indications after Regeneron opted out of further co-development and co-commercialization. However, Regeneron will be eligible for low double-digit royalties on mivelsiran sales if the drug is approved.
