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FDA Approves First Treatment for Rare Nerve Disease


August 10, 2018

The FDA has approved Onpattro (patisiran; Alnylam Pharmaceuticals, Inc.) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first treatment approved for patients with polyneuropathy caused by hATTR.

The approval of Onpattro is the first approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment. Within the body’s cells, RNA acts as a messenger carrying instructions from DNA for controlling the synthesis of proteins. This new drug class works by silencing a portion of RNA involved in causing the disease. Specifically, Onpattro encases the siRNA into a lipid nanoparticle to deliver the drug directly into the liver and alters or halts the production of disease-causing proteins.

Approval of Onpattro was based on a clinical trial that examined 225 patients. Of the 225 patients, 148 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. According to the findings, patients who received Onpattro had better outcomes on measures of polyneuropathy including muscle strength, sensation (pain, temperature, numbness), reflexes and autonomic symptoms (blood pressure, heart rate, digestion) compared to those receiving the placebo infusions. Further, this study cohort also scored better on assessments of walking, nutritional status and the ability to perform activities of daily living.

The most commonly reported adverse reactions included, infusion-related reactions including flushing, back pain, nausea, abdominal pain, dyspnea (difficulty breathing) and headache. 

The FDA granted Onpattro Fast Track, Priority Review, and Breakthrough therapy designations. Onpattro also received Orphan Drug designation. which provides incentives to assist and encourage the development of drugs for rare diseases.

Currently, hATTR affects approximately 50,000 worldwide.

Julie Gould


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