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 disappointing sales and uptake since its launch three years ago. The decision marks a significant shift for a product once hailed as a potential blockbuster and highlights the ongoing challenges in commercializing gene therapies.

Roctavian's Journey from Promise to Divestment

Roctavian, approved in Europe in 2022 and in the U.S. in 2023, was initially viewed as a groundbreaking treatment for hemophilia A. As a one-time, long-lasting therapy, it offered an alternative to chronic treatments and was expected to provide both clinical and economic benefits. However, the therapy's commercial performance has fallen far short of expectations.

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

Commercial Challenges and Industry Implications

The underwhelming performance of Roctavian reflects broader challenges in the gene therapy market:

• Sales figures: Roctavian generated only $3.5 million in product sales in 2023, $26 million in 2024, and $23 million in the first nine months of 2025.
• Market expectations: Initial projections by analysts, such as Leerink Partners, estimated peak sales of $2.2 billion. BioMarin's own early estimates for 2023 ranged from $50 million to $150 million.
• Industry-wide trend: Other companies have faced similar challenges. Pfizer stopped selling its hemophilia B gene therapy due to weak demand, and CSL's Hemgenix has also seen slow uptake.

Hardy previously cited the "complexity" of getting patients on treatment as a factor in Roctavian's performance. Additional challenges included concerns about the durability of the therapy's benefits and pricing issues that complicated reimbursement discussions.

Next Steps and Market Impact

BioMarin's decision to divest Roctavian represents a strategic realignment for the company. While the therapy will remain commercially available in the U.S., Germany, and Italy until next steps are finalized, this move signals a significant shift in the gene therapy landscape.

The company has committed to continuing monitoring and support for patients who have received Roctavian treatment. This decision aligns with BioMarin's portfolio strategy and, according to Hardy, "offers the most promising opportunity for ensuring continued patient access to Roctavian."

As the gene therapy market continues to evolve, BioMarin's experience with Roctavian serves as a cautionary tale for the industry, highlighting the complexities of bringing innovative treatments from scientific breakthroughs to commercial success.