FDA approves drug for treatment of acute hepatic porphyria

The vast majority of people with acute hepatic porphyria do not have frequent attacks; according to the National Organization for Rare Diseases, just 3%-5% of people fall into that category.

The FDA has granted the approval of Givlaari to Alnylam Pharmaceuticals.

Givosiran is the second-ever drug based on a Nobel prize-winning technique known as RNA interference, Alnylam’s .

The drug, which will be branded as Givlaari, will cost $575,000 per year, the company announced onWednesday; after discounts, it expects the average price to be $442,000 per year. Each vial of the drug will cost $39,000.

“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,” said Richard Pazdur, M.D., 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. “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 was based on the results of a clinical trial of 94 patients with acute hepatic porphyria. Patients received a placebo or Givlaari. Givlaari’s performance was measured by the rate of porphyria attacks that required hospitalizations, urgent health care visits or intravenous infusion of hemin at home. Patients who received Givlaari experienced 70% fewer porphyria attacks compared to patients receiving a placebo.

Common side effects for patients taking Givlaari were nausea and injection site reactions. Health care professionals are advised to monitor patients for anaphylactic (allergic) reaction and renal (kidney) function. Patients should have their liver function tested before and periodically during treatment.

The FDA granted this application Breakthrough Therapy designation and Priority Review designation. Givlaari also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

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