On Wednesday, the U.S. Food and Drug Administration announced the approval of Givlaari (givosiran) for the treatment of acute hepatic porphyria.
Previous treatment options for this genetic disorder have only provided partial relief from the pain of porphyria attacks, according to Richard Pazdur, M.D., director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA Center for Drug Evaluation and Research. Givlaari helps to reduce the number of attacks. The recommended dose of Givlaari is a subcutaneous injection of 2.5 mg/kg once monthly.
Approval of Givlaari was based on clinical data from 94 patients with acute hepatic porphyria. Compared with patients who received placebo, patients who received Givlaari had 70 percent fewer porphyria attacks that required hospitalization, urgent health care visits, or home intravenous infusion of hemin.
Commonly reported side effects of Givlaari include nausea and injection site reactions. Clinicians should monitor patients for anaphylactic reaction and renal function, and patients should have their liver function tested before and during treatment.
Approval was granted to Alnylam Pharmaceuticals.
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