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.”
Alnylam is one of several companies that have pursued development of RNAi treatments in recent years. Arrowhead Pharmaceuticals, for example, is in clinical phases of developing RNAi candidates ARO-HBV for hepatitis B and ARO-AAT for the liver disease associated with alpha-1 antitrypsin deficiency.
In June, Akcea Therapeutics won approval in Europe for Tagsedi (inotersen) for stage 1 or stage 2 polyneuropathy in adult patients with hATTR. Tegsedi is under FDA review with a target PDUFA decision date of October 6.
hATTR is a rare disease that affects about 50,000 people worldwide, characterized by the buildup of amyloid in the body’s organs and tissues that interferes with their normal functioning. Most frequently, the protein deposits most occur in the peripheral nervous system, which can result in a loss of sensation, pain, or immobility in the arms, legs, hands, and feet. Amyloid deposits can also affect the functioning of the heart, kidneys, eyes, and gastrointestinal tract. Treatment options have generally focused on symptom management.