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BioMarin Pharmaceutical Inc., a biotech company, has decided to adjust its ROCTAVIAN business by focusing on commercial operations in three markets: the United States, Italy, and Germany.1 The medicine is approved and reimbursed in these nations as a treatment for severe hemophilia A. BioMarin will be altering these ROCTAVIAN efforts by decreasing investments when it comes to development and manufacturing.
Being that the company produced enough commercial supply of ROCTAVIAN to sustain demand, BioMarin has placed its gene therapy manufacturing facility in an “idle” state until additional production is needed.
By limiting its focus on these aforementioned markets, annual direct ROCTAVIAN expenses will decrease to approximately $60 million starting next year, and this reduction plan is well underway. With this proactive approach, BioMarin anticpates profitability by end of 2025. Development in other markets will be conditional upon the progress that’s made in these markets.
“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, BioMarin’s president and CEO. “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.”
A study involving ROCTAVIAN has proven quite promising thus far, especially after four years.
“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,” commented Hank Fuchs, MD, the company’s president of worldwide research & development. “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.”
In other BioMarin news, the FDA has approved the company’s supplemental biologics license application (sBLA) for BRINEURA, its branded form of cerliponase alfa that slows the loss of the ability to walk independently in children of all ages with neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as tripeptidyl peptidase 1 (TPP1) deficiency.2 Prior to this approval, BRINEURA was indicated in symptomatic children 3 years of age and older with late infantile CLN2 disease. The updated, expanded indication now features children of all ages with CLN2 disease, regardless of whether they present symptoms or not.
“Today’s approval represents a significant step forward in enabling children to be treated with BRINEURA as early as possible, when we can have the greatest impact in altering the natural course of disease,” said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. “We know that every day counts for families affected by serious genetic conditions such as CLN2 disease, which is characterized by a rapid onset of neurodegenerative symptoms. We have been working diligently since BRINEURA’s initial approval to support this expanded use in children of all ages, even before they begin to show symptoms.”
References
1. BioMarin Announces Updated Strategy for ROCTAVIAN to Focus on U.S., Germany and Italy. BioMarin. August 5, 2024. Accessed August 6, 2024. https://investors.biomarin.com/news/news-details/2024/BioMarin-Announces-Updated-Strategy-for-ROCTAVIAN-to-Focus-on-U.S.-Germany-and-Italy/default.aspx
2. U.S. Food and Drug Administration Approves BioMarin’s BRINEURA® (cerliponase alfa) for Children Under 3 Years with CLN2 Disease. BioMarin. July 24, 2024. Accessed August 6, 2024. https://investors.biomarin.com/news/news-details/2024/U.S.-Food-and-Drug-Administration-Approves-BioMarins-BRINEURA-cerliponase-alfa-for-Children-Under-3-Years-with-CLN2-Disease/default.aspx