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FDA approves patisiran infusion for hereditary transthyretin-mediated amyloidosis
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The FDA announced that it has approved patisiran infusion for the treatment of patients with peripheral nerve disease caused by hereditary transthyretin-mediated amyloidosis.
The treatment (Onpattro, Alnylam Pharmaceuticals) is the first of its kind to be approved by the FDA for patients with polyneuropathy associated with hereditary transthyretin-mediated amyloidosis, and the first FDA approval for small interfering ribonucleic acid treatment, according to a press release from the agency.
Hereditary transthyretin-mediated amyloidosis is a genetic disease that produces buildup of abnormal amyloid protein in peripheral nerves, the heart and elsewhere in the body, the FDA stated in the release.
“There has been a long-standing need for a treatment for hereditary transthyretin-mediated amyloidosis,” Billy Dunn, MD, director of the division of neurology products for the FDA’s Center for Drug Evaluation and Research, said in the press release. “This unique targeted therapy offers these patients an innovative treatment for their symptoms that directly affects the underlying basis of this disease.”
Small interfering ribonucleic acids is a new class of drugs that silences the part of the RNA that causes the disease. The approved treatment is delivered to the liver as an infusion by encasing the small interfering ribonucleic acid into a lipid nanoparticle, according to the press release. The prevention of the protein transthyretin can reduce amyloid deposit accumulation in peripheral nerves, which can improve symptoms.
Efficacy of this treatment was shown in a randomized clinical trial with 225 patients. Those who received the infusion had improved outcomes and better assessments of nutritional status, walking and ability to perform activities of daily living compared with those who received placebo, according to the release.
“New technologies like RNA inhibitors that alert the genetic drivers of a disease have the potential to transform medicine so we can better confront and even cure debilitating illnesses,” Scott Gottlieb, MD, FDA commissioner, said in the press release. “We’re committed to advancing scientific principles that enable the efficient development and review of safe, effective and groundbreaking treatments that have the potential to change patients’ lives.”
Disclosures: Gottlieb is commissioner of the FDA. Dunn is an employee of the FDA.