Novel gene-silencing drug holds promise for this rare disease


FDA clears first-of-its-kind gene therapy for rare disease.

FDA okayed a first-of-its-kind gene silencing treatment to treat a rare disease.

Patisiran liquid complex injection (Onpattro, Alnylam Pharmaceuticals), is the first therapeutic utilizing RNA interference (RNAi) and only FDA-approved medication to treat the rare disease, polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. The treatment is available immediately.

“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,” said Akshay Vaishnaw, MD, PhD, president of R&D at Alnylam, in a statement.

The approval also holds promise for the broader RNAi treatment area. The novel therapy silences genes that are not functioning correctly.

HATTR amyloidosis is a rare, inherited, rapidly progressive and life-threatening disease with a constellation of manifestations. In addition to polyneuropathy, hATTR amyloidosis can lead to other significant disabilities including decreased ambulation with the loss of the ability to walk unaided, a reduced quality of life, and a decline in cardiac functioning.

In the largest controlled study of hATTR amyloidosis, 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.

“FDA approval of Onpattro represents an entirely new approach to treating patients with polyneuropathy in hATTR amyloidosis and shows promise as a new era in patient care,” said John Berk, MD, associate professor of medicine at Boston University School of Medicine and assistant director of the Amyloidosis Center at Boston University School of Medicine, in the Alnylam statement.

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