NEW YORK – BioMarin Pharmaceutical on Monday said it's adjusting its business strategy for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec), focusing its commercial operations on the US, Germany, and Italy and cutting other expenses.
The new strategy aims to overcome slow sales of the multimillion-dollar gene therapy with a goal of the product becoming profitable for BioMarin next year.
These changes 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," the company said in a statement.
San Rafael, California-based BioMarin expects the new strategy, which also involves reducing other investments in development and manufacturing, will lower annual direct expenses from Roctavian to about $60 million, beginning next year. The company said it 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," BioMarin President and CEO Alexander Hardy said in a statement. "By rightsizing our resourcing, we are creating a path for Roctavian to contribute to our profitability while still providing full support to patients."
Roctavian is a single-dose gene therapy that uses an adeno-associated virus to deliver a functional copy of the FVIII gene that encodes the protein responsible for blood clotting. Patients with hemophilia A, a condition caused by FVIII gene mutations, have a deficiency of the protein that can lead to uncontrolled bleeding episodes.
The gene therapy is approved and reimbursed in the three countries at the center of BioMarin's new strategy, earning approval in the US in 2023 with a list price of $2.9 million and in the EU in 2022 with a list price of €1.5 million ($1.6 million). BioMarin has justified the high price tag by saying it reduces the need for routine FVIII prophylaxis, which can cost about $800,000 per year for patients.
However, in Q2 2024, BioMarin treated only five patients with Roctavian. The gene therapy generated $7.4 million in revenue in Q2, from three US patients and two in Italy, and $8.3 million in the first six months of 2024.
To cut expenses, BioMarin has paused manufacturing for the gene therapy, since there's currently enough commercial supply of Roctavian on hand to serve the anticipated demand in the three countries. The company also will not enroll new participants into clinical development programs, though it will continue to support and monitor patients who received treatment through a clinical trial.
BioMarin said efforts to expand Roctavian into other markets will be dependent on its progress in the US, Germany, and Italy.