On February 23, 2026, BioMarin Pharmaceutical Inc. has announced that it has decided to pull the hemophilia A gene therapy ROCTAVIAN (valoctocogene roxaparvovec-rvox) from the market.
The decision follows the company’s announcement in October 2025 that it would explore options to divest ROCTAVIAN and seek a potential buyer with an established presence in hematology or gene therapy. Despite BioMarin’s efforts, the company was unable to identify a qualified buyer. This decision is not related to ROCTAVIAN’s efficacy or safety profile.
The gene therapy treatment will cease availability at the end of May 2026.
The company has stated that it will continue to support individuals who have already been treated with ROCTAVIAN and to provide ongoing monitoring and follow-up care. The company will maintain its commercial patient support program and honor existing outcomes-based agreements with payers.
ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis.
The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023.
The United States, Germany, and Italy are among the few countries where ROCTAVIAN has been launched and has granted reimbursement. In Italy, ROCTAVIAN is reimbursed under Class H (hospital distribution only) since January 23, 2024 and it has also been granted conditional innovation status. The P&R procedure lasted for about 62 weeks from the submission of AIFA P&R Dossier to publication of P&R Decree in the Italian Official Gazette.
In conclusion, this news shows that, although ROCTAVIAN represented a major scientific advancement, the market 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.
Not even the reimbursement was enough to ensure long-term market availability.
If you’d like to learn more about this topic or regarding the new drugs that have been approved for reimbursement in Italy over the past 7 years, please contact us at the following email address: valuebox@valuevector.com .
