U.S. FDA Approves Alnylam's Drug for Rare Genetic Disorder

By Reuters Staff

November 21, 2019

(Reuters) - The U.S. Food and Drug Administration said on Wednesday it had approved Alnylam Pharmaceuticals Inc's gene silencing drug as a treatment for patients with a rare inherited disorder.

The drug was approved to treat acute hepatic porphyria, a genetic disorder that can cause seizures and back pains. (http://bit.ly/37oqz9Z)

The drug, known as Givlaari (givosiran), uses RNA interference to target and "silence" specific genetic material, blocking the production of the deadly protein that causes the disease.