The EMA’s CHMP has adopted a positive opinion recommending approval of vutrisiran for hATTR amyloidosis in adult patients with stage 1 or 2 polyneuropathy. The EC’s decision is expected in Sept 2022
The opinion was based on the 18mos. results from the P-III (HELIOS-A) study evaluating vutrisiran in a ratio (3:1) in 164 patients at 57 sites in 22 countries. The trial met the 1EPs & 2EPs of study at 9 & 18mos. & showed the reversal in neuropathy impairment along with encouraging safety & tolerability profile
Vutrisiran is under ANVISA & PMDA review. If vutrisiran is approved by EC, it will be commercialized under the brand name Amvuttra as 1st treatment option to demonstrate reversal in neuropathy impairment with SC administration
Ref: Bussinesswire | Image: Alnylam
