FDA Approves Alynlam’s RNAi Therapeutic for hATTR Amyloidosis

Rare Daily Staff

The U.S. Food and Drug Administration approved Alnylam Pharmaceuticals’ Onpattro, a first-of-its-kind RNA interference therapeutic, for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

Hereditary transthyretin-mediated amyloidosis is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.

hATTR amyloidosis represents a major unmet medical need with significant morbidity and mortality, affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival of 3.4 years for patients presenting with cardiomyopathy.

Onpattro is an intravenously administered RNAi therapeutic targeting transthyretin in development for the treatment of hATTR amyloidosis. It is designed to target and silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. This may help to reduce the deposition and facilitate the clearance of TTR amyloid in peripheral tissues and potentially restore function to these tissues. It is the first and only FDA-approved treatment for this indication.

In clinical studies, Onpattro was shown to improve polyneuropathy – with reversal of neuropathy impairment in a majority of patients – and to improve a composite quality of life measure, reduce autonomic symptoms, and improve activities of daily living.

Onpattro was reviewed by the FDA under Priority Review and had previously been granted Breakthrough Therapy and Orphan Drug designations.

“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,” said John Maraganore, CEO of Alnylam. “With the potential for the sequential launches of several new medicines in the coming years, we believe we have the opportunity to meaningfully impact the lives of people around the world in need of new approaches to address serious diseases with significant unmet medical needs.”

Onpattro is expected to be available for shipment to healthcare providers in the U.S. within 48 hours. Alnylam is committed to helping people access the medicines they are prescribed and will be offering comprehensive support services for people prescribed Onpattro through Alnylam Assist.

August 10, 2018
Photo: John Maraganore, CEO of Alnylam