Amit Soni, MD, on Challenges to the Uptake of Gene Therapy for Hemophilia


The medical director of the Center for Inherited Blood Disorders discussed barriers to the widespread adoption of val-rox by the hemophilia A community.

This is the second part of an interview with Amit Soni, MD. For the first part, click here.

“I think by having more stories like ours get out in the community, that will further build confidence in the treatment so more patients and providers feel comfortable adopting this type of therapy.”

In November 2022, the FDA approved UniQure and CSL Behring’s etranacogene dezaparvovec, marketed as Hemgenix, which was the first gene therapy product to be approved in the United States for the treatment of hemophilia B.1 Less than a year later, the agency also approved BioMarin’s valoctocogene roxaparvovec, marketed as Roctavian, the first gene therapy approved in the US for the treatment of hemophilia A.2 Despite these momentous approvals, the uptake of these new one-time treatment options has been slow-paced, and many patients in the hemophilia community remain on previous standard of care treatment options. Notably, the first patient with hemophilia A, referred to as Maxwell L, was treated with val-rox in the real-world setting at the Center for Inherited Blood Disorders (CIBD) in Orange County California, in December 2023.3

Following Max’s treatment, CGTLive® sat down with Amit Soni, MD, the medical director of the CIBD, to learn more about some of the challenges to gene therapy’s adoption by patients with hemophilia. Soni pointed out that patients receiving val-rox are recommended to undergo lab draws once per week for the first 6 months posttreatment and once every 2 weeks for the next 6 months, in order to monitor for potential complications. Because of this commitment, a patient needs to be highly motivated in order to accept treatment with val-rox. Soni also brought attention to other difficulties, like obtaining coverage from insurance for the treatment and the need for special infrastructure like cleanrooms in centers that administer gene therapy products. In a separate comment after the interview, Soni also mentioned another barrier to treatment with val-rox: patients who have preexisting antibodies to adeno-associated virus 5 cannot receive it.

1. FDA approves first gene therapy to treat adults with hemophilia B. News release. FDA. November 22, 2022.
2. U.S. Food and Drug Administration Approves BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec-rvox), the First and Only Gene Therapy for Adults with Severe Hemophilia A. News release. BioMarin Pharmaceutical Inc. June 29, 2023. Accessed April 25, 2024.,-the-First-and-Only-Gene-Therapy-for-Adults-with-Severe-Hemophilia-A
3. Center for Inherited Blood Disorders Administers Country's First Gene Therapy Infusion to Treat Hemophilia A. News release. Center for Inherited Blood Disorders. January 11, 2024. Accessed April 25, 2024.
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