FDA Approves Patisiran Infusion for Hereditary Transthyretin-Mediated Amyloidosis
The FDA announced that it has approved patisiran infusion for the treatment of patients with peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis.
The treatment (Onpattro, Alnylam Pharmaceuticals) is the first of its kind to be approved by the FDA for patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis, and the first FDA approval for small interfering ribonucleic acid treatment, according to a press release from the agency.
Hereditary transthyretin-mediated amyloidosis is a genetic disease that produces buildup of abnormal amyloid protein in peripheral nerves, the heart and elsewhere in the body, the FDA stated in the release.
“There has been a long-standing need for a treatment for hereditary transthyretin-mediated amyloidosis,” Billy Dunn, MD, director of the division of neurology products for the FDA’s Center for Drug Evaluation and Research, said in the press release. “This unique targeted therapy offers these patients an innovative treatment for their symptoms that directly affects the underlying basis of this disease.”
