FDA approves Givlaari

Givlaari (givosiran), a treatment for adult patients with the genetic disorder acute hepatic porphyria, has been approved by the US FDA.

The FDA granted approval of the product based on the results of a clinical trial that was conducted on ninety-four patients with the genetic disorder. All of the patients were given either Givlaari or a placebo. The performance of Givlaari was determined by the amount of porphyria attacks that required patients to go to hospital, to need intravenous infusion of hemin at home or vital health care visits. Results showed that those patients who given the placebo experienced seventy-percent more porphyria attacks versus those who received Givlaari.

Patients who received Givlaari experienced common side effects of the treatment including injection site reactions and nausea, and the advice is that healthcare professionals stay vigilant for signs of kidney malfunction and anaphylactic reaction.

Acting Director of the Office of Oncologic Diseases in the FDA’s Centre for Drug Evaluation and Research, and Director of the FDA’s Oncology Centre of Excellence, Richard Pazdur, said: “This build up 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. 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.”