The FDA today granted Alnylam Pharmaceuticals approval for a first-in-class small interfering ribonucleic acid (siRNA) treatment—the first therapy indicated for polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adults.
Onpattro™ (patisiran) is an infusion treatment whose approval comes 20 years after the discovery of RNA interference. Onpattro is designed to interfere with RNA production of an abnormal form of the protein transthyretin (TTR). By preventing the production of TTR, the drug is intended to help reduce the accumulation of amyloid deposits in peripheral nerves, improving symptoms and helping patients better manage their polyneuropathy.
“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,” Alnlyam CEO John Maraganore, Ph.D., said in a statement. “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.”