By Sonia Amaral Rohter
Sanofi SA (>> Sanofi) and Alnylam Pharmaceuticals Inc. (>> Alnylam Pharmaceuticals, Inc.) reported Wednesday that Patisiran met all primary and secondary endpoints in a Phase III trial of its use against a rare, inherited disorder.
The trial looked at the use of Patisiran in patients with hereditary transthyretin-mediated amyloidosis, a progressively debilitating disease in which a gene mutation causes the production of a type of abnormal protein that accumulates in body organs and tissues, damaging them. Patisiran is designed to target and silence the mechanism that leads to production of this protein.
Patisiran is part of a class of medicines called RNA interference, or RNAi, therapeutics that aim to inhibit the expression of certain genes in order to treat or prevent disease.
Alnylam Chief Executive John Maraganore said: "We are very proud to report the first-ever positive Phase III results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines."
Alnylam said it would file a new drug application with the U.S. Food and Drug Administration in late 2017. Should the company receive regulatory approval, Alnylam will commercialize Patisiran in the U.S., Canada and Western Europe, while Sanofi Genzyme will commercialize the product in the rest of the world.
The full results of the trial will be presented on November 2.
Write to Sonia Amaral Rohter at [email protected]