BioMarin Pharmaceutical stated it will move to divest its gene therapy Roctavian, a one-time infusion treatment for adults with severe hemophilia A. This decision follows persistent underperformance in the product’s commercial rollout and reflects a strategic realignment toward priorities with greater growth momentum. Roctavian, despite being the first gene therapy of its kind to receive approval for the bleeding disorder, has struggled to gain traction since entering the market, hindered by reimbursement challenges and slow patient uptake.
The company will seek out-licensing or partnership opportunities for Roctavian, with the aim of maintaining access for patients currently treated in key markets, including the United States, Germany, and Italy. BioMarin will continue to support and monitor those who have received the therapy while it explores next steps for the asset. Leaders at BioMarin cited the need to focus resources on their enzyme therapies and skeletal condition treatments, such as Voxzogo, which have shown robust revenue growth and become more central to the organization’s commercial strategy.
Financial updates from BioMarin highlighted improved overall revenue in recent quarters, mainly driven by the success of other portfolio products. The company stated that pivots like the Roctavian divestment are part of ongoing efforts to maximize long-term growth by prioritizing assets with higher commercial promise. This strategic change signals BioMarin’s intent to recalibrate its pipeline and focus while ensuring continuity of care for current hemophilia A patients.
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