“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 Maxwell L., adult living with hemophilia A. “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.”

Hemophilia is a blood disorder typically passed from a parent to their child through genes. It is caused by a mutation or change in one of the genes that prevents the clotting protein from working properly or to be missing altogether – this can lead to spontaneous bleeding. If not diagnosed and treated early, hemophilia can lead to crippling pain, internal bleeding into muscles and joints, severe joint damage, disability, and even early death. Hemophilia A, the most common of the two types of hemophilia, is estimated to affect as many as 33,000 males in the U.S.¹ Until recent breakthroughs in gene therapy, the blood disorder was considered a lifelong condition with no cure – it could only be treated by replacing the missing clotting factor or its function in the blood. The one-time gene therapy infusion provided to the patient at the CIBD clinic is designed to address the underlying genetic cause of hemophilia A, offering the potential for long-term relief and a significant reduction in the need for traditional clotting factor replacement therapies.

“Our CIBD team is deeply honored to be part of Max’s journey as the first in the country to receive this newly FDA approved cutting-edge treatment for hemophilia A. Being able to infuse it here at his treatment center allows our care team to continue to work collaboratively to ensure patients not only manage their condition but achieve the highest quality of life possible,” said Amit Soni, MD, Medical Director, Center for Inherited Blood Disorders.

The patient was administered the FDA-approved infusion, Roctavian™, to treat hemophilia A on December 29, 2023, at CIBD. The gene therapy infusion was approved by the FDA on June 29, 2023. Max is now under close monitoring as the medical team tracks the therapy’s efficacy and potential on the management of hemophilia A. Anticipated to bring life-changing results, this new treatment is proven to decrease the frequency and severity of bleeding episodes for individuals living with hemophilia A. For more information about CIBD and the center’s ongoing initiatives, please visit https://cibd-ca.org/.

About the Center for Inherited Blood Disorders

The Center for Inherited Blood Disorders (CIBD) has cared for hundreds of children and adults in Southern California, offering a safety net clinic that provides health care services specifically to patients with inherited blood disorders. CIBD has been able to increase access to care for those who are economically challenged by providing medical care regardless of ability to pay. CIBD is also a national leader in directing federal initiatives to promote regional blood disorder networks that provide team-based clinical care and uniformly track health outcomes. Learn more by visiting www.cibd-ca.org.

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