BioMarin to Divest Hemophilia Gene Therapy Roctavian After Disappointing Sales

BioMarin Pharmaceutical announced Monday it will divest its hemophilia A gene therapy Roctavian, marking a dramatic reversal for a treatment once expected to revolutionize care for patients with the bleeding disorder and generate billions in revenue for the company.

In a statement accompanying the company's third-quarter earnings report, CEO Alexander Hardy said BioMarin will "pursue options to divest Roctavian and remove it from our portfolio" after nearly three years of disappointing commercial performance. While the company maintains that Roctavian "has an important role to play in the treatment of hemophilia A," it is now actively evaluating out-licensing options to transfer the product to another manufacturer.

"This decision is consistent with BioMarin's portfolio strategy and offers the most promising opportunity for ensuring continued patient access to Roctavian," Hardy added in the statement, suggesting the company believes the therapy may fare better under different ownership.

A Promising Beginning Meets Commercial Reality

The announcement represents a steep and swift fall from Roctavian's highly anticipated launch trajectory. The therapy's approval in Europe in 2022, followed by U.S. regulatory clearance in 2023, were hailed as significant scientific milestones—the culmination of years of intensive research into developing genetic medicines for hemophilia A, a genetic bleeding disorder affecting thousands of patients worldwide.

As a one-time, long-lasting treatment, Roctavian was positioned as a transformative alternative to the chronic therapies that people with hemophilia A typically rely on to prevent dangerous bleeding episodes. The gene therapy promised to provide sustained factor VIII production—the clotting protein missing in hemophilia A patients—potentially eliminating or dramatically reducing the need for regular infusions that have been the standard of care for decades.

Beyond its clinical promise, Roctavian was also held up as a clear demonstration of the potential economic value proposition of gene therapies. Despite carrying a high initial price tag, proponents argued it could prove cost-effective by alleviating the need for years of expensive supportive care, emergency treatments, and chronic therapy that patients would otherwise require throughout their lifetimes.

Blockbuster Expectations and Reality's Harsh Lesson

At the time of its approval, Wall Street analysts and industry observers widely viewed Roctavian as a blockbuster product in the making. Investment firm Leerink Partners projected peak sales of $2.2 billion, while BioMarin itself expressed significant optimism, initially forecasting the therapy would generate between $50 million and $150 million in revenue during 2023 alone.

The reality proved starkly different. Roctavian has instead become a cautionary tale illustrating the formidable challenges drugmakers can face when attempting to commercialize gene therapies, even those with strong scientific rationales and regulatory approval.

BioMarin was forced to quickly and sharply revise its revenue forecasts downward throughout 2023, ultimately recording just $3.5 million in product sales that year—a tiny fraction of initial projections. The situation improved only marginally in subsequent years, with the therapy accounting for merely $26 million in total revenue during 2024. Through the first nine months of 2025, Roctavian generated just $23 million in sales, the company disclosed Monday.

Multiple Barriers to Commercial Success

CEO Hardy has previously pointed to the "complexity" of getting patients enrolled and treated as a primary reason for Roctavian's underwhelming commercial performance. The process of identifying appropriate candidates, conducting necessary pre-treatment testing, coordinating infusion logistics, and arranging long-term monitoring proved more cumbersome than anticipated.

However, complexity wasn't the only obstacle. Emerging questions about the durability of Roctavian's therapeutic benefits created hesitation among both physicians and patients. If the gene therapy's effects might diminish significantly over time, the value proposition of choosing a one-time treatment over established chronic therapies became less clear-cut.

Additionally, Roctavian's substantial price tag made reimbursement negotiations with insurers and healthcare systems particularly challenging. Payers expressed concerns about budget impact and demanded extensive evidence of long-term value, slowing patient access even in cases where physicians recommended the treatment.

A Broader Industry Challenge

BioMarin's struggles with Roctavian are not an isolated phenomenon in the gene therapy space. The company finds itself in growing company among manufacturers facing similar commercialization headwinds.

Pharmaceutical giant Pfizer cited persistently weak demand when it chose to discontinue sales of its gene therapy for hemophilia B, a less common variant of the bleeding disorder. Meanwhile, CSL's Hemgenix, another hemophilia B gene therapy that received considerable attention at launch, has similarly experienced slow commercial uptake, falling well short of initial sales expectations.

These setbacks have prompted industry-wide discussions about the gap between the scientific promise of gene therapies and the practical realities of bringing them successfully to market. Issues including reimbursement challenges, patient identification and education, physician adoption patterns, and long-term efficacy monitoring have all emerged as significant hurdles.

Gene Therapy Market Continues Expansion Despite Setbacks

Despite these individual product struggles, the broader gene therapy sector continues to demonstrate robust growth and attract substantial investment. The global gene therapy market, valued at $7.21 billion in 2023, reached $8.85 billion in 2024 and is projected to advance at a resilient compound annual growth rate of 19.4% from 2025 through 2032, culminating in a forecasted valuation of $36.55 billion by the end of that period.

This continued market expansion reflects ongoing innovation in the field, with numerous gene therapies in development for conditions ranging from rare genetic disorders to more common diseases. Investors and pharmaceutical companies remain committed to the technology's long-term potential, even as they absorb lessons from early commercial disappointments.

Transition Plans and Patient Continuity

In last August, rather than immediately divesting Roctavian, BioMarin had opted to pare down spending on the product while maintaining its commercial presence. Hardy cited signs of launch progress in the three markets the company chose to prioritize: the United States, Germany, and Italy.

The company stated Monday it will continue making Roctavian commercially available in these three countries until "next steps are finalized" and a divestiture is completed. BioMarin also committed to providing ongoing monitoring and support services for patients who have already received the treatment, ensuring continuity of care during the transition period.

The company's announcement leaves open questions about which entity might acquire Roctavian and whether a new owner with different resources, expertise, or market approach might achieve better commercial results with the therapy. For now, BioMarin's decision underscores both the immense potential and substantial challenges that continue to define the gene therapy landscape.