FDA Approves Treatment for Rare Genetic Disorder

FDA announced on Nov. 20, 2019 that it has approved Givlaari (givosiran) for the treatment of acute hepatic porphyria, a genetic disorder that results in the buildup of toxic porphyrin molecules during the production of heme. The application was granted through the agency’s Breakthrough Therapy, Priority Review, and Orphan Drug designations.

A buildup of toxic porphyrin molecules causes attacks that can lead to severe pain, paralysis, respiratory failure, seizures, and changes in mental status, according to 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,” Pazdur stated in a press release.

The agency based the approval on the results of a clinical trial that involved 94 patients with the disorder. The study measured the performance of the drug by the rate of porphyria attacks that required hospitalization, urgent healthcare visits, or intravenous infusion of hema. According to FDA, 70% fewer attacks were seen in patients who received Givlaari compared to those that received a placebo.

Source: FDA