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FDA gives nod to Alnylam's Onpattro for peripheral nerve disease

The Food and Drug Administration has given its blessing for Alnylam Pharmaceuticals' Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis, or hATTR, in adult patients.

This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, which is a rare, debilitating and often fatal genetic disease that affects about 50,000 people worldwide. The disease is characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart and other organs. This is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid, or siRNA, treatment, according to the FDA.

This new class of drugs works by silencing a portion of RNA involved in causing the disease. Onpattro encases the siRNA into a lipid nanoparticle to deliver the drug directly into the liver, in an infusion treatment, to alter or halt the production of disease-causing proteins.

"This approval is part of a broader wave of advances that allow us to treat disease by actually targeting the root cause, enabling us to arrest or reverse a condition, rather than only being able to slow its progression or treat its symptoms. In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness and loss of mobility," FDA commissioner Scott Gottlieb said, in a press statement. "New technologies like RNA inhibitors, that alter the genetic drivers of a disease, have the potential to transform medicine, so we can better confront and even cure debilitating illnesses. 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."

"There has been a long-standing need for a treatment for hereditary transthyretin-mediated amyloidosis polyneuropathy. This unique targeted therapy offers these patients an innovative treatment for their symptoms that directly affects the underlying basis of this disease," director of the division of neurology products in the FDA's Center for Drug Evaluation and Research Billy Dunn, said, in a press statement.

The FDA granted this application 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.
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