First Gene Therapy for Severe Hemophilia A Is Approved

Roctavian, an adeno-associated virus vector-based gene therapy, was approved by the US Food and Drug Administration (FDA) for the treatment of adults with severe hemophilia A without pre-existing antibodies to adeno-associated virus serotype 5 detected by an FDA-approved test.

This approval comes after a safety and effectiveness trial showed that Roctavian improved participants’ annualized bleeding rate after 3 years.

Roctavian is given via injection and has been associated with mild changes in liver function, headache, nausea, vomiting, fatigue, abdominal pain, and infusion-related reactions.

“Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding,” said Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, via FDA press release.

“Today’s approval represents an important advance[ment] in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy.”

Reference:

FDA approves first gene therapy for adults with severe hemophilia A. Press Release. US Food and Drug Administration. June 29, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-adults-severe-hemophilia