BRUSSELS/FRANKFURT/PARIS (dpa-AFX) - RNAi therapeutics company Alnylam Pharmaceuticals, Inc. (ALNY) announced Tuesday that the European Commission has granted marketing authorization for AMVUTTRA (vutrisiran), an RNAi therapeutic for the treatment of hereditary transthyretin-mediated or hATTR amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy.
The EC approval is based on positive 18-month results from the HELIOS-A Phase 3 study. In the trial, AMVUTTRA significantly improved the signs and symptoms of hATTR amyloidosis. More than 50 percent of patients in the trial experienced halting or reversal of their polyneuropathy manifestations.
AMVUTTRA is the company's second RNAi therapeutic for hATTR amyloidosis.
HELIOS-A was a global, randomized, open-label, multicenter, Phase 3 study that evaluated the efficacy and safety of AMVUTTRA across a diverse group of patients with hATTR amyloidosis with stage 1 or stage 2 polyneuropathy.
In the study, AMVUTTRA met the primary and all secondary endpoints at both 9 months and 18 months, demonstrating reversal in neuropathy impairment and an encouraging safety and tolerability profile.
Results of the HELIOS-A study were published in Amyloid in July, 2022.
David Adams, Head of the Neurology department at Bictre hospital AP-HP, University of Paris-Saclay and Lead Investigator for the HELIOS-A Study, said, 'RNAi therapeutics are changing the future of medicine .. Results from the HELIOS-A study have shown the potential AMVUTTRA has to benefit patients with hATTR amyloidosis with stage 1 or stage 2 polyneuropathy, whilst also helping reduce treatment burden through subcutaneous dosing once every three months.'
Vutrisiran was previously granted Orphan Drug Designation in the European Union and U.S. for the treatment of ATTR amyloidosis and in Japan for transthyretin type familial amyloidosis with polyneuropathy.
In June 2022, AMVUTTRA was approved by the U.S. Food and Drug Administration for the treatment of the polyneuropathy of hATTR amyloidosis in adults. Vutrisiran is under review by the Brazilian Health Regulatory Agency and the Japanese Pharmaceuticals and Medical Devices Agency.
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