NEW YORK – BioMarin Pharmaceutical this week announced it would restructure into three business units as part of its goal of reaching $4 billion in revenue by 2027.
The San Rafael, California-based drugmaker's new corporate strategy now centers on three business units — skeletal conditions, enzyme therapies, and its hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec) — to reach that goal. It also expects $2.75 billion to $2.83 billion in revenue for 2024. For 2023, the company reported $2.4 billion in revenue.
The company last month announced changes to its business strategy for Roctavian, which has been experiencing slow sales. Roctavian, which was approved by the US Food and Drug Administration in 2023, is an adeno-associated virus-based gene therapy that delivers a functional copy of the FVIII gene, which encodes a key blood-clotting protein. Patients with hemophilia A, which is caused by FVIII gene mutations, are deficient in that protein, leading to uncontrolled bleeding episodes.
At that time, the company said it would focus its commercial operations on the US, Germany, and Italy, where the gene therapy is already approved, and cut other expenses with the aim of making the treatment profitable by the end of 2025. In the US, Roctavian has a list price of $2.9 million. Related to this strategic shift, BioMarin further laid off 225 employees.
According to the company, the bulk of its expected $4 billion in revenue will come from its skeletal condition unit, which includes its peptide analog treatment for achondroplasia, Voxzogo (vosoritide). Voxzogo is approved in the US for children with achondroplasia, a skeletal dysplasia caused by certain gain-of-function variants in the FGFR3 gene, and the company is also exploring Voxzogo in other genetic short stature conditions.
BioMarin is now targeting a potential approval for Voxzogo for hypochondroplasia, a milder short stature condition that is also primarily caused by FGFR3 variants, in 2027.