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FDA approves Givlaari, first treatment for acute hepatic porphyria

The FDA approved Givlaari for the treatment of adults with acute hepatic porphyria, a rare genetic disorder that leads to buildup of toxic porphyrin molecules that form during the production of heme.

“This buildup can cause acute attacks, known as porphyria attacks, which can lead to severe pain and paralysis, respiratory failure, seizures and mental status changes. These attacks occur suddenly and can produce permanent neurological damage and death,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in the FDA brief. “Prior to today’s approval, treatment options have only provided partial relief from the intense unremitting pain that characterizes these attacks. The drug approved today can treat this disease by helping to reduce the number of attacks that disrupt the lives of patients.”

The approval of Givlaari (givosiran, Alnylam) was based on results from a clinical trial of 94 patients with acute hepatic porphyria. Researchers randomly assigned patients to receive subcutaneous givosiran 2.5 mg/kg (n = 48) or placebo (n = 46) monthly for 6 months.

Patients who received givosiran experienced 70% fewer porphyria attacks compared with placebo.

Common side effects among those treated with givosiran included nausea and injection site reactions.

The FDA advised that health care professionals monitor patients for anaphylactic reaction and renal function. Patients should have their liver function tested before and periodically during treatment.

References:

www.fda.gov

Balwani M, et al. GS-14. Presented at: International Liver Congress; April 10-14, 2019; Vienna, Austria.