FDA Approves First Treatment for Rare Nerve Disease

The US Food and Drug Administration (FDA) has approved Onpattro (patisiran) infusion for the treatment of adults with peripheral nerve disease—or polyneuropathy—caused by hereditary transthyretin-mediated amyloidosis, which affects about 50,000 people worldwide.

Onpattro is not only the first FDA-approved treatment for this indication, but it is also the first FDA-approved drug in a new class called small interfering ribonucleic acid treatment.

The approval was granted following a trial of 225 patients who were randomly assigned to receive an Onpattro infusion (n = 148) or placebo infusion (n = 77) once every 3 weeks for 18 months.

Patients who were treated with Onpattro demonstrated better outcomes for muscle strength, sensation, reflexes, autonomic symptoms, walking, nutritional status, and ability to perform daily activities compared with those who received placebo.

Infusion-related reactions such as flushing, back pain, nausea, abdominal pain, dyspnea, and headache were the most commonly reported adverse reactions among Onpattro-treated patients.

—Christina Vogt

Reference:

FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease [press release]. US Food and Drug Administration. August 10, 2018. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/UCM616518.htm?utm_campaign=08102018_PR_FDA approves new drug for rare disease, hATTR&utm_medium=email&utm_source=Eloqua Accessed on August 10, 2018.