Biomarin fired 225 employees as part of a major restructuring effort. Credit: SOPA Images via Getty Images.

BioMarin Pharmaceuticals plans to let 225 staff members go as it reconfigures its Roctavian (valoctocogene roxaparvovec) development programme.

The company announced the reduction in workforce in a 28 August SEC filing, stating that the downsizing will play a role in its “organisational redesign efforts”. The company plans to substantially complete the structural change by the end of this year.

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The US-based company declared the layoffs soon after sharing news about several organisational shifts this month.

BioMarin appointed a new executive vice president and chief business officer and also announced a pivot in its commercial focus for its lead asset Roctavian to drive commercial operations in the US, Germany, and Italy. Regulators in the three countries have already approved and reimbursed the therapy for the treatment of severe haemophilia A.

The company had also proposed a reduction in Roctavian development and manufacturing investments. In a 5 August press release, BioMarin declared that the reduction in manufacturing programs is expected to reduce the annual direct Roctavian expenses to approximately $60m, beginning next year.

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The company has also chosen to stop enrolling patients into Roctavian clinical programmes, choosing to focus its efforts on treating the currently enrolled patients.

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Roctavian is a gene therapy, which acts by stimulating the production of clotting protein Factor VIII and replacing the mutant genes. This restores the body’s factor VIII plasma concentrations to adequate levels to allow for normal clotting in patients with haemophilia A.

Roctavian is the only FDA-approved gene therapy for haemophilia A. The list price for the gene therapy stands at $2.9m, making it the sixth most expensive drug in the world, as per a GlobalData analysis. This year, the therapy amassed $800,000 in Q1 2024 sales, thanks to the treatment of the first haemophilia patient with Roctavian in Italy. In 2023, the company earned $3.5m in Roctavian sales through the dosing of three patients. GlobalData predicts that sales for the therapy could reach $451m in 2030.

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