BioMarin Pharmaceutical is giving up on the hemophilia gene therapy Roctavian, announcing on Monday plans to offload a first-of-its-kind medicine once expected to become a future blockbuster.
In a statement announcing the company’s third-quarter earnings, CEO Alexander Hardy said BioMarin will “pursue options to divest Roctavian and remove it from our portfolio.” BioMarin still believes Roctavian “has an important role to play in the treatment of hemophilia A” and is evaluating “out-licensing options” as a result, Hardy said.
“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.
The announcement culminates what’s been a fast fall for Roctavian since its launch began three years ago.
Roctavian’s approval in Europe in 2022 and in the U.S. a year later were scientific milestones, the culmination of years of research developing a genetic medicine for hemophilia A. As a one-time, long-lasting treatment, Roctavian was billed as an alternative to the chronic therapies people with hemophilia A use to prevent bleeding. It was also seen as a clear example of the potential economic bargain of a gene therapy that, despite a high initial price tag, might alleviate the need for supportive care patients would receive instead.
At the time, many Wall Street analysts viewed the product as a blockbuster-to-be. Leerink Partners analysts once projected $2.2 billion in peak sales, and BioMarin was similarly optimistic, estimating early on that the therapy would generate anywhere from $50 million to $150 million in 2023.
Instead, Roctavian has become a cautionary tale of the challenges drugmakers can face selling a gene therapy. BioMarin quickly and sharply slashed its revenue forecasts for 2023 and ended up recording $3.5 million in product sales that year. The therapy accounted for only $26 million in 2024, and just $23 million over the first nine months of 2025, the company said Monday.
Hardy has previously cited the “complexity” of getting patients on treatment as a reason for Roctavian’s commercial performance. But doubts about the durability of its benefits and a price tag that made reimbursement discussions challenging also slowed its sales trajectory.
BioMarin wasn’t alone in reporting sluggish sales, either. Pfizer cited weak demand in choosing to stop selling a gene therapy for the less common hemophilia B. Uptake has also been slow for CSL’s Hemgenix, another hemophilia B gene therapy.
Last August, BioMarin pared down Roctavian spending rather than choosing to sell it altogether, with Hardy at the time citing signs of launch progress in the three countries — the U.S., Germany and Italy — the company chose to focus on. The company will still make Roctavian commercially available in those countries until the “next steps are finalized,” and continue to provide monitoring and support for people who receive treatment, it said Monday.
