BioMarin to Narrow Its Commercial Focus for Hemophilia A Gene Therapy

BioMarin recently announced that they will be changing the scope of their efforts to manufacture and market their gene therapy product ROCTAVIAN.™ The product was first approved in the summer of 2023 for the treatment of adults with severe hemophilia A without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved diagnostic test.

In a new press release the company signaled their plan to limit commercial operations to the three countries where ROCTAVIAN is both “approved and reimbursed as treatment for hemophilia,” including the United States, Germany, and Italy. Any future expansion into additional markets will be contingent upon commercial success in the aforementioned countries. In the meantime, a moratorium on manufacturing of the therapy has been put in place as current supply is projected to meet demand. Beginning next year, BioMarin will also be reducing its direct expenses associated with ROCTAVIAN.

“We continue to believe that ROCTAVIAN is an important option for people with severe hemophilia A, offering the potential for years of bleed control after a single, one-time treatment,” said Alexander Hardy, President and Chief Executive Officer of BioMarin. “By rightsizing our resourcing, we are creating a path for ROCTAVIAN to contribute to our profitability while still providing full support to patients. We are deeply grateful to the hemophilia community and to the patients and healthcare providers who participated in our clinical trials, and we will continue to provide support, as well as to meet our regulatory commitments for ongoing monitoring.”

Despite the relatively modest uptake of the therapy to date, BioMarin has cited more recent progress in “facilitating access” to ROCTAVIAN at hemophilia treatment centers, securing reimbursement agreements with insurers, and generating patient interest.

No new patients will be enrolled in ROCTAVIAN clinical trial programs. However, BioMarin will continue to monitor and support participants who have already received a one-time infusion of the product. This will also include the ongoing collection of long-term efficacy and safety data.

Source: BioMarin press release dated August 5, 2024