Press release
Alnylam Receives Positive CHMP Opinion for Vutrisiran for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult Patients with Stage 1 or Stage 2 Polyneuropathy
- Positive Opinion Based on HELIOS-A Phase 3 Study - - European Commission Decision Expected in September 2022 - CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n- Positive Opinion Based on HELIOS-A Phase 3 Study -\n\n- European Commission Decision Expected in September 2022 -\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending approval of vutrisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated (hATTR) amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. If approved by the European Commission (EC), vutrisiran will be commercialized under the brand name AMVUTTRA™ as the first and only treatment option to demonstrate reversal in neuropathy impairment with subcutaneous administration once every three months.\n\n“Today’s decision by the CHMP is good news for patients living with hATTR amyloidosis with polyneuropathy, a rare, rapidly progressive and fatal condition for which there are few treatment options. We expect a European Commission (EC) decision on approval in September 2022. If approved, vutrisiran will become Alnylam’s second treatment for hATTR amyloidosis to be commercialized in Europe, delivering on our commitment to bring continued innovation to patients. We believe vutrisiran provides a compelling efficacy and safety profile, as well as the benefit of a quarterly subcutaneous dosing regimen, to help manage this debilitating condition,” said Kasha Witkos, SVP, Head International Region at Alnylam.\n\nThe CHMP decision was based on positive Month 18 results from HELIOS-A, a global, randomized, open-label, multicenter, Phase 3 study that evaluated the efficacy and safety of vutrisiran across a diverse group of patients with hATTR amyloidosis with stage 1 and stage 2 polyneuropathy. Vutrisiran met the primary and all secondary endpoints of the study at both 9 months and 18 months, demonstrating reversal in neuropathy impairment and an encouraging safety and tolerability profile. After 18 months of dosing, the frequently occurring adverse reactions in vutrisiran-treated patients were arthralgia (joint stiffness) and pain in extremity (pain in arms and legs). Other adverse reactions reported with vutrisiran were dyspnea (shortness of breath), injection site reaction and an increase in blood alkaline phos...