BioMarin 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...

Strategic Focus will Reduce Operating Expenses with the Goal of Achieving ROCTAVIAN Profitability by End of 2025

BioMarin to Provide Continued Monitoring and Support to Those Treated in Clinical Trials and Commercially

SAN RAFAEL, Calif. , Aug. 5, 2024 /PRNewswire/ -- 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

Research & Development

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.

Forward-Looking Statements

BioMarin ® and ROCTAVIAN ® are registered trademarks of BioMarin Pharmaceutical Inc.

Founded in 1997, BioMarin is a global biotechnology company dedicated to transforming lives through genetic discovery. The company develops and commercializes targeted therapies that address the root cause of genetic conditions. BioMarin's robust research and development capabilities have resulted in multiple innovative commercial therapies for patients with rare genetic disorders. The company's distinctive approach to drug discovery has produced a diverse pipeline of commercial, clinical, and pre-clinical candidates that address a significant unmet medical need, have well-understood biology, and provide an opportunity to be first-to-market or offer a substantial benefit over existing treatment options. For additional information, please visit www.biomarin.com .

SOURCE BioMarin Pharmaceutical Inc.