First hemophilia A patient in Europe treated with commercial Roctavian

Treatment became available in US following FDA approval this summer

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer.

“Today represents an important milestone for the hemophilia community and for patients and physicians around the world seeking access to Roctavian,” Jeff Ajer, executive vice president and chief commercial officer at BioMarin, said in a company press release.

Dozens of people in Germany are undergoing screening to see if they’re eligible for Roctavian, according to BioMarin.

Final price negotiations for the therapy are ongoing with federal officials in Germany and BioMarin is working with officials in France and Italy on agreements on pricing, reimbursement, and other launch conditions so people there can also access it. 

“The burden of severe hemophilia A for people who are living with the condition is substantial and there remains a significant unmet need for effective treatments that do not require chronic therapy. This one-time infusion represents an important milestone, offering new hope and potential for eligible individuals in Germany,” said Robert Klamroth, MD, PhD, a physician at the Center for Hemophilia and Hemostaseology at the Vivantes Klinikum in Berlin, Germany. “Bringing this therapy to all those who can benefit is critical to improve outcomes for individuals with severe hemophilia A.”

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Access to Roctavian

Roctavian became available in the U.S. following its approval by the U.S. Food and Drug Administration (FDA) this summer. Hemophilia treatment centers in the U.S. are screening patients to determine eligibility and there’s been an influx of patient consent forms and warranty agreements that are expected to help facilitate access to the therapy, according to the company.

“We look forward to more people gaining access to Roctavian in the rest of Europe as well as the United States, where Roctavian recently received FDA approval,” Ajer said.

Roctavian is a one-time gene therapy that delivers a working copy of the gene encoding factor VIII (FVIII) — a blood clotting protein that’s defective or missing in hemophilia A — to liver cells where most clotting proteins are made. By delivering a healthy copy of the gene, the therapy would restore the ability to make functional FVIII, thereby preventing excessive bleeding.

In clinical trials, most patients given Roctavian didn’t have any clinically relevant bleeds after up to three years of follow-up. The gene therapy carries a list price of $2.9 million.

The gene therapy was approved to treat adults with severe hemophilia A who don’t have antibodies against AAV5, the viral vector Roctavian uses to deliver its genetic payload. Last year, the therapy was granted conditional approval in Europe for adults with severe hemophilia A who don’t have detectable antibodies against AAV5 or inhibitors, which are neutralizing antibodies against FVIII that can reduce standard replacement therapies’ effectiveness.