Alnylam Pharma: Patisiran Phase 3 Study Meets Endpoints - Quick Facts

(RTTNews) - Alnylam Pharmaceuticals, Inc. (ALNY) and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint and all secondary endpoints. The primary endpoint was the change from baseline in the modified neuropathy impairment score at 18 months. The key secondary endpoint was improvement in quality of life. The overall safety profile of patisiran was encouraging.

The APOLLO trial enrolled 225 hATTR amyloidosis patients with polyneuropathy, representing 39 genotypes, at 44 study sites in 19 countries around the world.

Based on the positive results, Alnylam expects to file its first New Drug Application in late 2017 and first Marketing Authorisation Application shortly thereafter. Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil and other countries, to begin in the first half of 2018. Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.