BioMarin Pharma (BMRN) Announces Updated Strategy for ROCTAVIAN to Focus on U.S., Germany and Italy

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced it will make changes to its ROCTAVIAN^® business to focus commercial operations on three markets, the United States, Germany and Italy, where the medicine is approved and reimbursed as a treatment for severe hemophilia A. BioMarin will adjust its ROCTAVIAN efforts across the company, reducing additional investments in development and manufacturing.

By focusing commercial, research and manufacturing programs, BioMarin anticipates reducing annual direct ROCTAVIAN expenses to approximately $60 million, beginning in 2025. The company has already begun to operationalize the reduction of ROCTAVIAN expenses this year to achieve $60 million in expenses beginning in full-year 2025. As a result of these changes, the company expects ROCTAVIAN to be profitable by the end of 2025.

"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."

Hank Fuchs, M.D., President of Worldwide Research & Development continued, "We continue to be impressed by the durable and sustained bleed control demonstrated following treatment with ROCTAVIAN based on more than 500 patient years of observation in our pivotal program. At year four in that study, 82% of people who participated remain off prophylaxis, a significant therapeutic achievement when considering the high burden of chronic treatment for people with severe hemophilia A."

The updated approach to ROCTAVIAN will include the following changes:

Commercial & Manufacturing

• BioMarin will focus on the U.S., Germany and Italy, where ROCTAVIAN is approved and reimbursed. The company is encouraged by recent progress in facilitating access to ROCTAVIAN at hemophilia treatment centers.
• This includes successful execution of single patient agreements with key insurers and the readiness of hemophilia treatment centers in the U.S., advancing discussions with German sub-insurers to allow coverage for those interested in ROCTAVIAN, and encouraging patient interest and access in Italy.
• Expansion into other markets will be dependent on progress in the U.S., Germany and Italy.
• With ample commercial supply of ROCTAVIAN on hand to serve anticipated demand, the company has placed the gene therapy manufacturing facility in an idle state until such time when additional production is necessary.

Research & Development

• The company will not enroll new participants in clinical development programs but will continue to support patients who have already received ROCTAVIAN treatment, generate long-term safety and efficacy data from previously enrolled studies, and fulfill the regulatory commitments related to its clinical programs.

These strategic updates will allow BioMarin to ensure that resources are being used on the medicines, approved and in development, that are most likely to have the greatest impact for patients, while continuing to ensure that ROCTAVIAN is available for people with severe hemophilia A.

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