FDA approves first treatment for hereditary ATTR amyloidosis

The U.S. Food and Drug Administration today approved Patisiran, a novel therapy for the treatment of hereditary ATTR amyloidosis polyneuropathy. This approval comes after positive results from the readout of the Patisiran clinical trial APOLLO – published July 5, 2018 in The New England Journal of Medicine (NEJM) – in which patients on Patisiran had statistically significant improvement in neuropathy and quality of life measures over those on placebo. Patisiran will be commercialized under the brand name Onpattro.

“Hereditary ATTR amyloidosis is an aggressive, rapidly progressing, debilitating and fatal disease, and the need for treatment options is urgent.” Says Dr. Vaishali Sanchorawala, Director of the Amyloidosis Center at Boston University School of Medicine and Boston Medical Center, “The robust evidence provided by the APOLLO phase 3 trial showed significant improvement in motor, sensory and autonomic neuropathy, a significant reduction in disease symptoms, and favorable safety profile compared to placebo.”

Patisiran is an RNA interference drug that works by reducing the production of the protein transthyretin (TTR), which is the culprit protein misfolding into amyloid in patients with hATTR. It is given through an intravenous (IV) infusion along with pre-drugs every three weeks.

“Patisiran is designed to target and silence specific messenger RNA, blocking the production of TTR protein before it is made. This can enable the clearance of TTR amyloid deposits in the tissues and organs and potentially can restore function.” Sanchorawala explains, “The therapeutic landscape of ATTR amyloidosis is evolving and expanding rapidly. The innovation and approval of Patisiran is an epic moment in this rare and underdiagnosed disease. I am honored to be a witness to this monumental time for the FDA approval for the first-in-class treatment of ATTR amyloidosis. Very exciting time for all the families with devastating ATTR amyloidosis.”

“It is exciting to see the release of patisiran for patients with hATTR and peripheral neuropathy.” Says Dr. Angela Dispenzieri, Professor of Medicine and Laboratory Medicine and Pathology at Mayo Clinic, “Although the registration trial was designed to test the drug’s efficacy in patients with hATTR peripheral neuropathy, post-hoc analyses also demonstrated improved outcomes for patients with hATTR cardiomyopathy in addition to hATTR peripheral neuropathy!”

Following FDA approval, Patisiran can be prescribed for treatment of hATTR polyneuropathy once the pharmaceutical company completes their preparations to bring the drug to market.

View more information about Patisiran here.

Read more about hereditary ATTR amyloidosis here.

Read original press release from FDA here.