The approval was based on the 9mos. results from the P-III (HELIOS-A) study evaluating Amvuttra vs patisiran in a ratio (3:1) in 164 patients with hATTR amyloidosis with polyneuropathy. The product is expected to be available in early July
The trial met the 1EPs & 2EPs i.e., improvement in mNIS+7 from baseline @9mos. (2.2point vs 14.8 point), 50% experienced an improvement in neuropathy impairment relative to baseline, Norfolk QoL-DN score & 10-MWT
Improvements in exploratory EPs, and efficacy results were consistent with 9mos. data with no drug-related discontinuations or deaths. The company provides Alnylam Assist, a patient support program that helps patients access Amvuttra, verifies insurance benefits & provides financial assistance to qualified patients
Ref: Bussinesswire | Image: Alnylam
