Alnylam announced the U.S. FDA has approved Givlaari (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). Givlaari was reviewed by the FDA under Priority Review and was granted Breakthrough Therapy and Orphan Drug Designations in the U.S. Givlaari is expected to be available for shipment to healthcare providers in the U.S. by year-end.
The FDA approval of Givlaari was based on positive results from the phase 3 ENVISION study. The randomized, double-blind, placebo-controlled, multinational study enrolled 94 patients with AHP at 36 study sites in 18 countries. The patients on Givlaari experienced 70% fewer porphyria attacks compared to placebo. Givlarri also resulted in a similar reduction in intravenous hemin use, as well as reductions in urinary aminolevulinic acid (ALA), and urinary porphobilinogen (PBG). The most common adverse reactions (reported in at least 20% of patients) were nausea and injection site reactions.
Givlaari is currently being reviewed under accelerated assessment by the European Medicines Agency (EMA) for the treatment of patients with AHP, after receiving Priority Medicines (PRIME) Designation and Orphan Drug Designation from the EMA.
About Givlaari (givosiran)
Givlaari is an RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1). Givlaari is Alnylam’s first commercially available therapeutic based on its Enhanced Stabilization Chemistry ESC-GalNAc conjugate technology to increase potency and durability. Givlaari works by specifically reducing elevated levels of aminolevulinic acid synthase 1 (ALAS1) messenger RNA (mRNA), leading to reduction of toxins associated with attacks and other disease manifestations of AHP.
About Acute hepatic porphyria
Acute hepatic porphyria (AHP) refers to a family of ultra-rare, genetic diseases characterized by potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. AHP is comprised of four types: acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), variegate porphyria (VP), and ALA dehydratase-deficiency porphyria (ADP). Each type of AHP results from a genetic defect leading to deficiency in one of the enzymes of the heme biosynthesis pathway in the liver.