Alnylam reports positive results from HELIOS-A phase III study of Vutrisiran in patients with hATTR amyloidosis with polyneuropathy

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Alnylam Pharmaceuticals has announced that the HELIOS-A phase III study of Vutrisiran, an investigational RNA interference (RNAi) therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints at nine months in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy.

According to a press release from the company, the primary endpoint was the change from baseline in the modified Neuropathy Impairment Score (mNIS+7) at nine months as compared to historical placebo data from the APOLLO phase III study of patisiran. The two secondary endpoints were changes in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) and gait speed assessed by the timed 10-meter walk test (10-MWT) compared to historical placebo. Vutrisiran met the primary endpoint (p<0.001) and achieved statistically significant results (p<0.001) for each of the Norfolk QoL-DN and 10-MWT secondary endpoints. In addition, Vutrisiran treatment showed improvement compared to placebo on the exploratory cardiac biomarker endpoint, NT-proBNP (nominal p<0.05). Vutrisiran also demonstrated an encouraging safety and tolerability profile.

Based on these positive results, the company plans to submit a New Drug Application (NDA) for Vutrisiran with the US Food and Drug Administration (FDA) in early 2021, and to follow with regulatory filings in additional countries, such as Brazil and Japan. The company plans to submit a Marketing Authorisation Application (MAA) in the EU upon obtaining the results of the 18-month analysis—expected in late 2021—as previously aligned with the European Medicines Agency (EMA). Vutrisiran has already been granted Orphan Drug Designation in the USA and the EU, and a Fast Track designation in the USA.


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