Rare Daily Staff

Alnylam and its partner Sanofi Genzyme announced positive late-stage results for patisiran, an experimental RNAi therapy to treat a painful and debilitating rare genetic nerve disease.

The positive results represent the first for an RNAi therapeutic in a late-stage study and sets the stage for Alnylam before year-end to file an application with to U.S. Food and Drug Administration to Patisiran as a treatment for hereditary ATTR amyloidosis with polyneuropathy.

“This moment is the culmination of a 15-year journey of tireless work by countless contributors who have overcome enormous scientific and business challenges to make RNAi therapeutics a reality,” said John Maraganore, CEO of Alnylam. “This is an incredibly exciting milestone for Alnylam and RNAi, and most importantly for patients and their treating physicians and families.”

Patisiran is an experimental RNAi therapeutic that uses the body’s natural processes to lower the levels of the TTR protein that causes TTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. Alnylam said this may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues.

The patisiran and placebo arms had similar frequencies of adverse events and serious adverse events. The frequency of deaths in the study was similar in the patisiran and placebo arms. Adverse events reported with greater frequency in the patisiran users compared to placebo included peripheral edema (29.7 percent vs. 22.1) and infusion-related reactions (18.9 percent vs. 9.1 percent), both of which were generally mild-to-moderate in severity.

Elias Zerhouni, president of global R&D for Sanofi, said there’s an urgent need for additional treatment options for patients with hATTR amyloidosis with polyneuropathy. “This is a significant milestone that supports our belief that RNAi therapeutics have the potential to become an innovative new class of medicines for patients with rare genetic diseases.”

Based on the results, Alnylam expects to file its first a marketing application with the FDA in late 2017 and one with European regulators 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 United States, Canada, and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.

Full results, including data from an exploratory analysis of the subgroup of patients with cardiac involvement, will be presented at the 1st European ATTR Amyloidosis Meeting for Patients and Doctors, on November 2, 2017 in Paris, France.

September 20, 2017

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