NEW YORK – BioMarin Pharmaceutical is laying off 225 employees in connection with a strategic shift the firm has initiated due to slow sales of its hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec).
In an 8K filing with the US Securities & Exchange Commission on Wednesday, the San Rafael, California-based drugmaker said the global workforce reduction was "in connection with organizational redesign efforts as well as [its] updated strategy for Roctavian and its decision to discontinue development of BMN 293." BioMarin's gene therapy BMN 293 was in preclinical development for myosin binding protein C3 hypertrophic cardiomyopathy.
On Aug. 5, BioMarin outlined that it will focus its commercial operations in the US, Germany, and Italy, and cut other development and manufacturing expenses, aiming to lower its annual direct expenses for Roctavian to around $60 million starting in 2025 and recognize profits from the gene therapy, which carries a multimillion-dollar price tag, by the end of next year.
US regulators approved Roctavian in 2023, which carries a list price of $2.9 million, and in the EU, the gene therapy was approved the prior year and carries a list price of €1.5 million ($1.7 million). BioMarin has said that the high price tag reflects the therapy's ability to reduce the need for routine FVIII prophylaxis, which can cost patients about $800,000 per year.
The present workforce reduction, BioMarin estimated, will incur a one-time cash expenditure of between $30 million and $35 million during the second half of 2024. The company notified employees impacted by layoffs on Wednesday and said it expects workforce reductions to be completed by year-end.