BioMarin to Divest Hemophilia A Gene Therapy Roctavian Amid Ongoing Sales Struggles
BioMarin Pharmaceutical has announced plans to divest its hemophilia A gene therapy, Roctavian, following persistently low sales and commercial challenges. The decision marks a significant shift for the company and highlights the complexities surrounding the commercialization of gene therapies in the pharmaceutical industry.
Roctavian's Journey from Promise to Divestiture
Roctavian, once hailed as a potential blockbuster, received approval in Europe in 2022 and in the U.S. in 2023. Despite its groundbreaking status as the first gene therapy for hemophilia A, the treatment has faced significant hurdles in market adoption.
BioMarin CEO Alexander Hardy stated in a recent earnings release, "We continue to believe Roctavian has an important role to play in the treatment of hemophilia A and are therefore evaluating out-licensing options for this innovative gene therapy." The company aims to ensure continued patient access to Roctavian through this divestiture process.
Commercial Challenges and Market Performance
The commercial trajectory of Roctavian has been far from the initial projections:
- 2023 sales: $3.5 million (significantly below initial estimates of $50-150 million)
- 2024 total sales: $26 million
- 2025 year-to-date sales: $23 million (as of Q3)
These figures starkly contrast with earlier analyst predictions, such as Leerink Partners' projection of $2.2 billion in peak sales.
Industry-Wide Implications for Gene Therapies
BioMarin's experience with Roctavian is not isolated. Other companies in the hemophilia gene therapy space have faced similar challenges:
- Pfizer ceased sales of its hemophilia B gene therapy due to weak demand
- CSL's Hemgenix, another hemophilia B gene therapy, has also seen slow uptake
These developments underscore the broader challenges in commercializing gene therapies, including complex patient treatment processes, concerns about long-term efficacy, and reimbursement hurdles associated with high price tags.
References
- BioMarin resigned to ending journey with hemophilia A gene therapy Roctavian, seeks divestiture amid sales struggle
After weighing other options amid a continued sales struggle, the company will look to take the only approved hemophilia A gene therapy off its plate two years after its launch.
- BioMarin, following sluggish sales, to offload hemophilia gene therapy
The company intends to divest Roctavian and “remove it from our portfolio,” CEO Alexander Hardy said, after nearly three years of slow uptake for a medicine once viewed as a future blockbuster.
Explore Further
What are the key terms or collaboration model being considered for the out-licensing of Roctavian?
What are the main factors contributing to the slow market uptake of Roctavian compared to initial sales projections?
How does Roctavian's clinical efficacy and safety data compare to competing hemophilia gene therapies such as CSL's Hemgenix?
Are there competitors in the gene therapy space pursuing similar divestiture or out-licensing strategies for their products?
What are the reimbursement challenges specific to Roctavian and other high-priced gene therapies in the pharma industry?