Givosiran
Givosiran, sold under the brand name Givlaari, is for the treatment of adults with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme. Givosiran is a small interfering RNA directed towards 5-aminolevulinic acid synthase, an important enzyme in the production of heme.History
In November 2019, givosiran was approved in the United States for the treatment of adults with acute hepatic porphyria.
The U.S. Food and Drug Administration granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation. The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals.
