Drugs

FDA Approves Alnylam’s Onpattro for Nerve Disease

FDA has approved an Alnylam Pharmaceuticals NDA for Onpattro (patisiran) infusion for treating peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA).

“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,” FDA commissioner Scott Gottlieb is quoted in a release as saying. “In this case, the effects of the disease cause a degeneration of the nerves, which can manifest in pain, weakness and loss of mobility. 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.”

siRNAs work by silencing a portion of RNA involved in causing the disease. “More specifically, 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,” the agency says. “Onpattro is designed to interfere with RNA production of an abnormal form of the protein transthyretin (TTR). By preventing the production of TTR, the drug can help reduce the accumulation of amyloid deposits in peripheral nerves, improving symptoms and helping patients better manage the condition.”

Approval was based on data from a clinical trial involving 225 patients, 148 of whom 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. Patients who received Onpattro had better outcomes on measures of polyneuropathy including muscle strength, sensation, reflexes and autonomic symptoms (blood pressure, heart rate, digestion) compared to those receiving the placebo, FDA says. The most common adverse reactions reported by patients treated with Onpattro are infusion-related reactions including flushing, back pain, nausea, abdominal pain, dyspnea (difficulty breathing) and headache, it says.

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