BioMarin Pharmaceutical Inc. has announced that it has decided to pull the hemophilia A gene therapy ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the United States market. This is following an announcement in October of 2025 that it was looking for a buyer for the therapy. A letter to the community, received by the National Bleeding Disorders Foundation (NBDF) on February 23, 2026, stated that the company would continue to make ROCTAVIAN available to the U.S. community through the end of May. It will also be available in Italy and Germany on the same timeline.
“As we navigate this transition, we are working closely with healthcare providers to support patient care and enable those who wish to receive treatment to do so,” the letter said. “We remain dedicated to ensuring that people who have already been treated receive ongoing monitoring and support. As part of this, we will continue our commercial patient support programs as well as honor our outcomes-based agreements with payers. While we work with global health authorities on next steps, we are committed to proactively and transparently communicating with the community about our plans.”
ROCTAVIAN was developed as the first gene therapy for adults with severe hemophilia A. Clinical development began in the mid-2010s. In August 2022, the therapy received conditional approval from the European Medicines Agency, allowing the drug to be legally marketed in countries in the European Union. ROCTAVIAN received US Food and Drug Administration (FDA) approval on June 29th, 2023. The result of decades of research into gene therapy for the treatment of hemophilia, this was a scientific milestone that was hailed as a great leap forward in treatment for hemophilia A.
Although ROCTAVIAN represented a major scientific advancement, access was difficult: its one-time high cost, insurance and reimbursement challenges, patient qualification, clinical monitoring requirements, and uncertainty about how long factor VIII expression would last all influenced decision-making and approval by payers.
The progress achieved through gene therapy research underscores how far treatment for hemophilia A has advanced. “We understand that people may feel disappointed in this particular decision,” said Nathan Schaefer, senior vice president of public policy and access at NBDF. “But each step forward in treatment contributes to a deeper understanding of durability, safety, access, and lived experience. NBDF will continue to encourage new treatments and innovations in care for the bleeding disorders community.”
The letter to the community from BioMarin can be read here.
