(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.
Cite this: U.S. FDA Approves Alnylam's Drug for Rare Genetic Disorder - Medscape - Nov 21, 2019.
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