BioMarin Pharmaceutical announced Monday it will divest Roctavian, its gene therapy for hemophilia A, marking the end of what was once projected to be a blockbuster treatment for the rare bleeding disorder. The company plans to explore out-licensing options while continuing to make the therapy available in the U.S., Germany, and Italy.
The decision represents a dramatic reversal for a therapy that achieved regulatory milestones as the first gene therapy approved for hemophilia A in Europe in 2022 and the U.S. in 2023. Roctavian was designed as a one-time treatment for adults with severe hemophilia A, a rare genetic disorder where blood doesn't clot properly due to lack of factor VIII protein.
Commercial Performance Falls Short of Expectations
Roctavian's commercial launch struggled significantly, generating just $3.5 million in sales in 2023 and approximately $26 million in 2024. These figures fell far short of BioMarin's initial projections of $100 million to $200 million for 2023, which the company later reduced to $50 million to $100 million, and eventually to less than $10 million.
The therapy's $2.9 million price tag created substantial barriers to market access. CEO Alexander Hardy previously cited the "complexity" of getting patients on treatment as a key factor in the therapy's commercial performance. Additional challenges included limited patient eligibility, slow reimbursement processes, and questions about the durability of treatment benefits.
Wall Street analysts had initially viewed Roctavian as a potential blockbuster, with Leerink Partners projecting peak sales of $2.2 billion. The therapy was positioned as an economic bargain despite its high upfront cost, potentially eliminating the need for chronic supportive care that hemophilia A patients typically require.
Investor Pressure Influences Strategic Direction
The weak performance attracted attention from activist investor Elliott Investment Management, which acquired a stake exceeding $1 billion in BioMarin in November 2023. Elliott successfully negotiated the appointment of three new independent directors and the formation of a strategic review committee to evaluate the company's operations.
"This decision is consistent with BioMarin's portfolio strategy and offers the most promising opportunity for ensuring continued patient access to Roctavian," Hardy stated in announcing the divestiture plans. The company emphasized it still believes Roctavian "has an important role to play in the treatment of hemophilia A."
Broader Gene Therapy Market Challenges
Roctavian's struggles reflect wider challenges in the gene therapy sector, particularly for rare disease treatments. BioMarin was not alone in experiencing disappointing sales in the hemophilia gene therapy space. Pfizer discontinued sales of its hemophilia B gene therapy due to weak demand, while CSL's Hemgenix, another hemophilia B gene therapy, has also experienced slow uptake.
The setback has prompted BioMarin to refocus on its more profitable business units, including enzyme therapies and treatments for skeletal conditions. The company's experience with Roctavian serves as a cautionary tale about the complexities of commercializing gene therapies, despite their scientific achievements and therapeutic potential.
The divestiture decision underscores the ongoing challenges pharmaceutical companies face in translating breakthrough gene therapies into commercial success, particularly in rare disease markets where patient populations are limited and reimbursement pathways remain complex.
