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What type of drug is Onpattro?

Medically reviewed by Judith Stewart, BPharm. Last updated on July 6, 2020.

Official Answer

by Drugs.com

Onpattro (patisiran) is the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatments.

Onpattro is used for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults. This form of polyneuropathy is a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in nerves, the heart and other organs.

Onpattro works by encasing siRNA in lipids and stops the production of disease-causing amyloid proteins.

Onpattro is administered via intravenous infusion over approximately 80 minutes, every 3 weeks.

Common side effects with Onpattro include flushing, back pain, nausea, abdominal pain, difficulty breathing, and headache.

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