FDA Approves Givosiran for Adult Patients with Acute Hepatic Porphyria

The FDA has approved givosiran (Givlaari, Alnylam Pharmaceuticals) for adult patients with acute hepatic porphyria (AHP), a genetic disorder resulting in the buildup of toxic porphyrin molecules formed during the production of heme, which binds oxygen in the blood, according to a press release.¹

The approval of givosiran was based on the results of a clinical trial which included 94 patients with AHP who received either a placebo or givosiran., Givosiran’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 givosiran experienced 70% fewer porphyria attacks compared with patients receiving a placebo, according to the FDA.¹

Common adverse events for patients taking givosiran were nausea and injection site reactions. The FDA advises health care professionals to monitor patients for anaphylactic reaction and renal function. Patients should have their liver function tested before and periodically during treatment.¹

AHP refers to a family of rare genetic disease characterized by potentially life-threatening attacks, and chronic debilitating symptoms that negatively impact daily functioning and quality of life for some patients. Not all patients that have a genetic mutation for AHP will develop symptoms and sudden attacks are associated with widespread dysfunction within the nervous system and a wide array of symptoms which can mimic other diseases, according to Pinpoint AHP.²

Porphyrin buildup can cause acute attacks, which can lead to severe pain and paralysis, respiratory failure, seizures and mental status changes, according to 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.¹

“These attacks occur suddenly and can produce permanent neurological damage and death,” Dr Pazdur said in a statement.¹ “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 FDA has granted Alnylam’s application Breakthrough Therapy designation and Priority Review designation. Additionally, the givosiran also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.¹

References

• FDA approves first treatment for inherited rare disease. FDA website. Published November 20, 2019. http://s2027422842.t.en25.com/e/es?s=2027422842&e=277676&elqTrackId=376c7bc788024cd5a73d955f2e3dcbdc&elq=60a6d32b837b41328e070e5c45b1a366&elqaid=10358&elqat=1. Accessed November 20, 2019.
• AHP is a rare genetic disease. Pinpoint AHP website. https://www.porphyria.com/about?gclid=EAIaIQobChMI1Ia0rLj55QIVipOzCh1mJAwnEAAYASABEgLSHPD_BwE&gclsrc=aw.ds. Accesed November 20, 2019.