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…



Read More Here

Be the first to comment on "Alnylam’s gene silencing drug wins FDA approval"

Leave a comment

Your email address will not be published.


*