FDA approves Givlaari to treat acute hepatic porphyria.- Alnylam Pharma

Alnylam Pharmaceuticals, Inc. has announced that the FDA approved Givlaari (givosiran) injection for subcutaneous use for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, potentially life-threatening attacks and, for some patients, chronic manifestations that negatively impact daily functioning and quality of life. Long-term complications of AHP can include chronic neuropathic pain, hypertension, chronic kidney disease and liver disease.

Givlaari was shown to significantly reduce the rate of porphyria attacks that required hospitalizations, urgent healthcare visits or IV hemin administration at home.The FDA approval of Givlaari was received in less than four months after acceptance of the NDA, and was based on positive results from the ENVISION Phase III study, a randomized, double-blind, placebo-controlled, multinational study of 94 patients with AHP, at 36 study sites in 18 countries � the largest ever interventional study conducted in AHP.

In ENVISION, AHP patients on Givlaari experienced 70% (95% CI: 60%, 80%) fewer porphyria attacks compared to placebo. Givlaari also resulted in a similar reduction in intravenous hemin use, as well as reductions in urinary aminolevulinic acid (ALA), and urinary porphobilinogen .