NBDF monitors emerging therapies and changes in the therapeutic landscape that may affect people living with bleeding disorders. One of those developments involves BioMarin’s decision to out-license (allowing another company to market and sell) ROCTAVIAN, their gene therapy for adults with severe hemophilia A.
This statement from BioMarin explains their decision:
“After careful consideration, we have decided to explore options to out license ROCTAVIAN (valoctocogene roxaparvovec) with another company and remove it from our portfolio of therapies. We believe that ROCTAVIAN could benefit from being part of a company with a strong presence in hematology and/or capabilities in gene or cell therapy, and that this is the best way to support our goal of ensuring this important treatment option reaches those who want to receive it.
This decision is not related to ROCTAVIAN’s safety or effectiveness, and we will continue to support individuals who are in the process of receiving treatment and that will continue as planned for any scheduled or upcoming ROCTAVIAN infusions.
During this time, we will maintain the same level of support we have always provided. We will continue to make ROCTAVIAN available in the U.S., Italy and Germany until next steps are finalized. We remain committed to supporting and monitoring all people treated with ROCTAVIAN. We are at the beginning of this process and will share more information as it becomes available.
We continue to believe that ROCTAVIAN has an important role in treating severe hemophilia A, and we remain dedicated to supporting this community.”
NBDF will continue to monitor developments related to ROCTAVIAN and other therapies for hemophilia.
