Alnylam Pharmaceuticals has secured approval from the US Food and Drug Administration (FDA) for its RNA interference (RNAi) therapeutic, Onpattro (patisiran) lipid complex injection, to treat polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
Onpattro is claimed to be the first-of-its-kind RNAi therapeutic and only FDA-approved treatment for this indication.
Earlier, Onpattro secured breakthrough therapy and orphan drug designations from the FDA.
The treatment uses a novel approach to target and reduce production of the TTR protein in the liver through the RNAi pathway.
The hATTR amyloidosis is a rare, inherited and life-threatening disease, and will also cause significant disabilities such as decreased ambulation with the loss of the ability to walk unaided, reduced quality of life and decline in cardiac functioning.
Alnylam CEO John Maraganore said: “Alnylam was founded on the vision of harnessing the potential of RNAi therapeutics to treat human disease, and this approval heralds the arrival of an entirely new class of medicines.
“We believe today draws us ever-closer to achieving our Alnylam 2020 goals of becoming a fully integrated, multi-product biopharmaceutical company with a sustainable pipeline.”
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.
Alnylam has randomized patients in a 2:1 ratio to receive intravenous Onpattro (0.3 mg per kg of body weight) or placebo once every three weeks for 18 months.
The study demonstrated that Onpattroimproved measures of polyneuropathy, quality of life, activities of daily living, ambulation, nutritional status and autonomic symptoms relative to placebo in adult patients with hATTR amyloidosis with polyneuropathy.
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.
Alnylam R&D president Akshay Vaishnaw said: “Today’s historic approval marks the arrival of a first-of-its kind treatment option for a rare and devastating condition with limited treatment options.”