Alnylam’s gene silencing drug wins FDA approval

(Reuters) – Alnylam Pharmaceuticals Inc’s drug for a rare hereditary disease won U.S. regulatory approval on Friday, becoming the first approved treatment from a new class of medicines that use gene silencing technology.

Alnylam’s patisiran, commercially named Onpattro, was approved to treat polyneuropathy in patients with hereditary ATTR amyloidosis, a potentially fatal condition that affects an estimated 50,000 people worldwide.

Patients with the disease have a genetic mutation that prevents a type of protein from keeping its normal structure, instead causing it to fold into an…

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