BioMarin to Divest Hemophilia Gene Therapy Roctavian Amid Slow Sales

BioMarin Pharmaceutical has announced plans to divest Roctavian, its gene therapy for hemophilia A, following nearly three years of disappointing sales performance. The decision marks a significant shift for a product once hailed as a potential blockbuster in the pharmaceutical industry.

Roctavian's Journey from Promise to Divestment

Roctavian, approved in Europe in 2022 and in the U.S. in 2023, represented a milestone in hemophilia treatment as a one-time, long-lasting gene therapy. Initially projected to generate between $50 million and $150 million in 2023, with some analysts predicting peak sales of $2.2 billion, the therapy has fallen far short of expectations.

BioMarin CEO Alexander Hardy stated in the company's third-quarter earnings report, "We will pursue options to divest Roctavian and remove it from our portfolio." Despite this decision, Hardy maintained that Roctavian "has an important role to play in the treatment of hemophilia A" and that the company is exploring "out-licensing options" to ensure continued patient access.

Commercial Challenges and Industry Implications

The therapy's commercial performance has been lackluster since its launch. BioMarin recorded only $3.5 million in Roctavian sales for 2023, followed by $26 million in 2024, and $23 million in the first nine months of 2025. Hardy previously cited the "complexity" of getting patients on treatment as a factor in the slow uptake.

Roctavian's struggles are not unique in the hemophilia gene therapy space. Pfizer recently halted sales of its hemophilia B gene therapy due to weak demand, and CSL's Hemgenix, another hemophilia B treatment, has also seen slow adoption.

Future of Hemophilia Gene Therapies

BioMarin's decision to divest Roctavian raises questions about the future of gene therapies in hemophilia treatment. The company plans to continue making Roctavian available in the U.S., Germany, and Italy until "next steps are finalized," and will maintain monitoring and support for patients who have received the treatment.

This development serves as a cautionary tale for the pharmaceutical industry, highlighting the challenges in commercializing advanced therapies despite their scientific promise. As the landscape for hemophilia treatments continues to evolve, the industry will be closely watching how this divestment unfolds and its implications for future gene therapy developments.