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

• 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.   

Forward-Looking Statements

This press release contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including, without limitation, statements about: BioMarin's updated strategy for ROCTAVIAN, including (i) expectations regarding reduction of annual direct ROCTAVIAN expenses beginning in 2025, (ii) expectations regarding ROCTAVIAN being profitable by the end of 2025, (iii)  the commercialization of ROCTAVIAN for the treatment of severe hemophilia A in the U.S., Germany and Italy, and potential expansion into markets beyond such countries, (iv) expectations regarding the supply of ROCTAVIAN and BioMarin's ability to meet product demand, (v) BioMarin's plans regarding research and development, including clinical development of ROCTAVIAN and continued support for patients, and (vi) other anticipated benefits, including BioMarin's ability to use its resources in a manner 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. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others: BioMarin's success in the commercialization of ROCTAVIAN, including achieving adequate market share and reimbursement levels; impacts of macroeconomic and other external factors on BioMarin's operations; results and timing of current studies and clinical trials and the release of data from those trials; the content and timing of decisions by the Food and Drug Administration, the European Commission and other regulatory authorities; actual sales of ROCTAVIAN; and those factors detailed in BioMarin's filings with the Securities and Exchange Commission, including, without limitation, the factors contained under the caption "Risk Factors" in BioMarin's Quarterly Report on Form 10-Q for the quarter ended March 31, 2024 as such factors may be updated by any subsequent reports. Stockholders are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

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

About BioMarin

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.