Biomarin plans to sell rights to hemophilia A therapy Roctavian
Expensive treatment will continue to be available in U.S., 2 other countries
Biomarin Pharmaceutical is looking to sell off rights to Roctavian (valoctocogene roxaparvovec-rvox), its approved gene therapy for hemophilia A.
“As we focus on the business units aligned with our strategic priorities, today we are announcing the decision to pursue options to divest Roctavian and remove it from our portfolio,” Alexander Hardy, president and CEO of Biomarin, said in a company press release.
Roctavian is currently approved in the U.S. and Europe for the treatment of certain adults with severe hemophilia A. Last year, Biomarin announced that, in an effort to make the therapy more profitable, it was limiting sales of Roctavian to three countries: Italy, Germany, and the U.S.
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 said it plans to continue making Roctavian commercially available in these three countries until the next steps are finalized. Biomarin said it hopes to license the therapy to another company, which would take over commercialization.
“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,” Hardy said. “This decision is consistent with BioMarin’s portfolio strategy and offers the most promising opportunity for ensuring continued patient access to Roctavian.”
Roctavian led to long-term bleed reductions in clinical trials
Hemophilia A is caused by mutations in the F8 gene, which provides instructions to make the clotting protein factor XIII (FXIII). People with hemophilia have reduced levels and/or activity of this protein, resulting in symptoms that include easy and prolonged bleeding.
Roctavian is designed to deliver a healthy version of the F8 gene to cells in the liver, where clotting factors are produced, thus allowing the body to make a functional FXIII protein that can help control bleeding. The one-time gene therapy is administered by an infusion into the bloodstream.
Clinical trials have demonstrated that Roctavian can lead to long-term reductions in bleeds and limit the need for standard hemophilia treatments. However, the therapy carries a substantial price tag, with a list price of nearly $3 million in the U.S. Biomarin has previously reported challenges in getting insurers to reimburse the cost of the therapy, which the company said has made it difficult for interested patients to access Roctavian.
Biomarin isn’t the only company that’s struggled to make a profit on hemophilia gene therapy. Earlier this year, Pfizer discontinued its hemophilia B gene therapy Beqvez (fidanacogene elaparvovec-dzkt), citing limited interest in gene therapies among the hemophilia community.
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