Press release
Alnylam Receives Approval in Europe for AMVUTTRA® (vutrisiran) for the Treatment of Hereditary Transthyretin-mediated (hATTR) Amyloidosis in Adult Patients with Stage 1 or Stage 2 Polyneuropathy
– AMVUTTRA Demonstrated Halting or Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months – – Decision Follows Positive
About this update from Alnylam Pharmaceuticals, Inc.
[{"type":"text","content":"\n– AMVUTTRA Demonstrated Halting or Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months –\n\n– Decision Follows Positive Opinion from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2022 –\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAlnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the European Commission (EC) has granted marketing authorization for AMVUTTRA® (vutrisiran), an RNAi therapeutic for the treatment of hereditary transthyretin-mediated (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, where AMVUTTRA significantly improved the signs and symptoms of hATTR amyloidosis, with more than 50 percent of patients experiencing halting or reversal of their polyneuropathy manifestations.\n\n“On behalf of patients across Europe, we could not be prouder to announce the approval of AMVUTTRA, our second RNAi therapeutic for hATTR amyloidosis. We are committed to delivering continued innovation to people affected by rare, life-limiting conditions where high unmet need still exists. Today’s decision now means we can progress working with health authorities across Europe to achieve responsible and sustainable access arrangements that allow us to bring AMVUTTRA to patients as quickly as possible. Our thanks go to the patients, families, investigators and study staff who have enabled us to reach this significant milestone,” said Kasha Witkos, SVP, Head International Region at Alnylam.\n\n“Although the considerable research into hATTR amyloidosis over the past few years has resulted in a more positive outlook for those diagnosed with the condition, there remain unmet needs in treatment for patients living with this rapidly progressive, multi-system disease”, said Professor David Adams, Head of the Neurology department at Bicêtre hospital AP-HP, University of Paris-Saclay and Lead Investigator for the HELIOS-A Study. “RNAi therapeutics are changing the future of medicine and I am honored to have contributed to these research efforts that have enabled us to bring an innovative new medicine to patients. Results from the HELIOS-A study have shown the potential AMVUTTRA has to benefit patient...