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FDA Approves Drug for Rare Peripheral Nerve Disease

FDA Web site; 2018 Aug 10

The US Food and Drug Administration (FDA) has approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized 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 (siRNA) treatment.

Approval of Onpattro was granted to Alnylam Pharmaceuticals, Inc., headquartered in Cambridge MA.

Dosage/administration: For patients weighing <100 kg, the recommended dosage is 0.3 mg/kg every 3 weeks by intravenous infusion. For patients weighing ≥100 kg, the recommended dosage is 30 mg. Patients should be premedicated with a corticosteroid, acetaminophen, and

antihistamines. The dosage should be filtered and diluted prior to administration and infused over approximately 80 minutes.

Adverse reactions: The most common adverse reactions reported by patients treated with Onpattro are infusion-related reactions including flushing, back pain, nausea, abdominal pain, dyspnea (difficulty breathing), and headache.


FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease. [news release]. Cambridge, MA: Alnylam Pharmaceuticals, Inc. August 10, 2018. Accessed August 20, 2018.