WFH statement - EN

WFH statement on discontinuation of valoctocogene roxaparvovec-rvox (ROCTAVIAN)

In October 2025, Biomarin announced its decision to remove valoctocogene roxaparvovec-rvox (ROCTAVIAN) from its product portfolio. On February 23, 2026, the company announced that due to the inability to find a buyer for the product, it will voluntarily withdraw valoctocogene roxaparvovec-rvox from the market. The World Federation of Hemophilia (WFH) acknowledges this outcome and encourages BioMarin and other gene therapy developers to continue monitoring the safety of discontinued products and to preserve relevant clinical data to support ongoing learning and patient safety.

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It’s imperative that pharmaceutical companies maintain their commitment to safety even after therapies are discontinued.  It is equally important that clinical trial and commercial data on all gene therapy products continue to be collected over the long-term to monitor safety outcomes.

The position of the WFH and other organizations has been outlined in two previously published articles:

  • “Critical Juncture in the Development of Hemophilia Therapies A Statement and Call-to-Action from the International Society on Thrombosis and Haemostasis (ISTH) European Association for Haemophilia and Allied Disorders (EAHAD) World Federation of Hemophilia (WFH) for the importance of continued research into and development of effective and safe hemophilia treatments” which can be read here
  • “The Critical Need to Consolidate All Gene Therapy Data in Haemophilia” which was published in Haemophilia and which can be read here

The WFH urges all pharmaceutical companies involved in hemophilia treatment products to maintain their commitments to our global community. Data can be collected through the WFH Gene Therapy Registry (GTR), a prospective, observational, and longitudinal registry designed to collect long-term data on people with hemophilia (PWH) who receive gene therapy. To find out more about the registry, click here.

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Disclaimer

The information on the WFH website is provided for general information purposes only. The WFH does not engage in the practice of medicine and under no circumstances recommends particular treatment for specific individuals. For diagnosis or consultation on a specific medical problem, the WFH recommends that you contact your physician or local treatment centre. Before administering any products, the WFH urges patients to check dosages with a physician or hemophilia centre staff, and to consult the pharmaceutical company’s printed instructions.

While every effort has been made to ensure the accuracy of the information on this site, the WFH does not guarantee the information is accurate, and is not responsible in any way whatsoever for damages arising out of the use of this website or any of the information contained herein.

Messages posted to WFH discussion forums, Facebook, Twitter, and other social media platforms do not represent the opinions of the World Federation of Hemophilia, its staff, or Board of Directors. The author of a message is solely responsibility for its content. Information posted on WFH social networks and platforms should never be a substitute for individualized professional medical advice, even when the author has medical qualifications or is considered an authority. Information posted to a discussion group should not be used to diagnose or treat a specific health problem without consulting a qualified healthcare professional. The WFH recommends that you contact your physician or local treatment centre if you have any individual questions or concerns.

References and links to other websites or references to other organizations, products, services, or publications do not constitute endorsement or approval by the WFH. The WFH is not responsible and assumes no liability for the content of any linked websites.

Fraud Alert

Unauthorized solicitations – Warning

The WFH has been made aware of various correspondences—circulated via e-mail and telephone—coming from individuals impersonating WFH staff or falsely stating that they are associated with the WFH. These correspondences, which may seek to obtain money using the name of someone affiliated with the WFH, are fraudulent and come from outside of our organization.

If you receive a suspicious solicitation, exercise extreme caution. In the case of an email, look at the email address to see if it looks suspicious (for example, all WFH emails come from @wfh.org).

We are asking you to remain vigilant, and if you have any doubts about the correspondence, please forward the email to the WFH at [email protected] or call +1 514-875-7944.