Therapy Areas: Cardiovascular
The US Food and Drug Administration approves Alnylam Pharmaceuticals' Onpattro
14 August 2018 -

The US Food and Drug Administration (FDA) has approved United States-based Alnylam Pharmaceuticals' RNA interference therapeutic, Onpattro (patisiran) lipid complex injection, intended to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, it was reported yesterday.

The product is claimed to be the first-of-its-kind RNAi therapeutic and only FDA-approved treatment for this indication. The product had previously received breakthrough therapy and orphan drug designations from the US FDA.

The approval was based on results from the randomized, double-blind, placebo-controlled and global phase 3 Apollo study. In the study, the safety and efficacy of Onpattro was assessed in a diverse global population of hATTR amyloidosis patients in 19 countries, with a total of 39 TTR mutations. The primary endpoint of the trial was the modified neuropathy impairment Score +7 (mNIS+7), which evaluated motor strength, reflexes, sensation, nerve conduction and postural blood pressure.

The hATTR amyloidosis is a rare, inherited and life-threatening disease that will also cause significant disabilities including decreased ambulation with the loss of the ability to walk unaided, reduced quality of life and decline in cardiac functioning.