FDA Approves First Gene Therapy for Severe Hemophilia A
The U.S. Food and Drug Administration on Thursday approved a costly single-dose gene therapy for patients with severe hemophilia A, a life-threatening hereditary bleeding disorder.
The treatment is not cheap: Roctavian will cost $2.9 million for a single infusion, the Associated Press reported.
“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,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in an agency news release. “Today’s approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy.”
For patients, this means a potential reduction in uncontrolled bleeding. Left untreated, severe hemophilia A can cause life-threatening bleeding into vital organs, including the kidneys and brain.
