Posted by Michael Wonder on 11 Aug 2018
FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease
10 August 2018 - The U.S. FDA today approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis in adult patients.
This is the first FDA-approved treatment for patients with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis a rare, debilitating and often fatal genetic disease characterised by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. It is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid treatment.
The efficacy of Onpattro was shown in a clinical trial involving 225 patients, 148 of whom were randomly assigned to receive an Onpattro infusion once every three weeks for 18 months, and 77 of whom were randomly assigned to receive a placebo infusion at the same frequency.
