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FDA Approves RNA-Interference Nerve Disease Therapy

Onpattro

(Alnylam Pharmaceuticals Inc.)

10 August 2018. A treatment that silences the actions of RNA and reverses damage to nerves from a rare inherited neurological disease was approved by the Food and Drug Administration. The drug, brand-named Onpattro by its developer Alnylam Pharmaceuticals Inc. in Cambridge, Massachusetts, is the first small interfering RNA therapy approved by FDA, according to the agency.

Onpattro is designed to treat peripheral nerve disease, affecting nerves in the body other than the brain or spinal cord, from hereditary transthyretin-mediated amyloidosis, or hATTR. The disorder results from accumulations of amyloid protein fibers on peripheral nerves in tissues and organs, leading to loss of sensation or pain in the limbs, hands, and feet, but also life-threatening disruptions of heart, kidney, eye, and gastrointestinal tract functions. According to FDA, some 50,000 individuals worldwide are affected by hATTR.

Up to now, the only treatments relieved symptoms of the disorder. Onpattro aims to prevent production of the transthyretin proteins that accumulate as amyloid fibers on peripheral nerves, thus reversing progression of the disease. The therapy works by interfering with ribonucleic acid, or RNA, providing instructions from the genes to produce the errant transthyretin proteins. RNA interference is a natural process for silencing genes in the body. Onpattro simulates this process with small interfering RNAs carried by lipid, or natural oil nanoscale particles, infused directly to the liver where the proteins are made.

FDA based its approval in part on results from a late-stage clinical trial of 225 patients with hATTR, randomized about 2 to 1 to receive Onpattro or a placebo. Patients receiving Onpattro showed better recovery from peripheral nerve disease on indicators such as strength, sensation, and reflexes, as well as indicators of organ-related functions including blood pressure, heart rate, and digestion. The most common adverse reactions were related to infusions, such as flushing, back pain, nausea, abdominal pain, and headache, but some trial participants also reported vision problems and a decrease in serum vitamin A levels.

“This approval,” says FDA Commissioner Scott Gottlieb in an agency statement, “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.” In addition to orphan drug status, Alnylam also received accelerated review of Onpattro under FDA’s Fast Track, Priority Review, and Breakthrough Therapy programs.

Alnylam also says the company is negotiating value-based agreements with some health insurers for Onpattro. Under value-based agreements, patients pay for the drug’s ability to deliver outcomes similar to those demonstrated in clinical trials.

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