Patient Receives First Hemophilia A Gene Therapy Outside a Clinical Trial

Roctavian™ was approved in the summer of 2023 for the treatment of adults with severe hemophilia.

The first hemophilia A gene therapy infusion to be delivered outside of a clinical trial setting was recently given to a patient in California.

The patient, an adult with severe hemophilia A, received a single infusion of Roctavian™ (BioMarin) at the Center for Comprehensive Care & Diagnosis of Inherited Blood Disorders (CIBD) in Orange, CA. Roctavian is currently the only hemophilia A gene therapy approved by the U.S. Food and Drug Administration (FDA). It received approval last summer for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved diagnostic test.

“This is more than a medical breakthrough; it’s a game-changer for me and others living with severe hemophilia A, particularly when considering I was able to drive myself to and from the clinic and did not have to stay overnight in a hospital,” said the therapy recipient. “Receiving this treatment is liberating – I am filled with hope and gratitude knowing treatments finally exist that will allow individuals like me to truly break free from the constraints of this disease and embrace life to the fullest.”

The care team at CIBD will continue to monitor the patient’s response to the therapy.

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Source: Businesswire, January 11, 2024, National Bleeding Disorders Foundation, January 2024

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