Roctavian, hemophilia A gene therapy, likely to soon arrive in US

Marketing in European countries expected by year's end

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin.

BioMarin also is working with authorities in Germany, Italy, and France to secure market access to Roctavian for patients in those countries.

“U.S. commercial launch activities are well underway following the June 29 approval [of Roctavian], in parallel with launch progress across a number of European countries,” Jean-Jacques Bienaimé, chairman and CEO of BioMarin, said in a company press release.

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Roctavian is a one-time therapy designed to deliver a healthy version of the gene encoding factor VIII (FVIII) — the blood clotting protein whose defect causes hemophilia A — to cells in the liver, the body’s main producers of blood clotting factors.

By allowing the body to make a working version of this clotting protein, the therapy is expected to reduce bleeds and the need for FVIII replacement therapy. Its list price will be $2.9 million.

The gene therapy was approved in the U.S. to treat adults with severe hemophilia A who don’t have antibodies against AAV5 or inhibitors to FVIII replacement therapies. AAV5 is the viral carrier used to deliver the therapy’s genetic payload, while inhibitors are antibodies against FVIII, which can reduce the efficacy of treatments.

“We were … very pleased to have received the highly anticipated U.S. approval of Roctavian, the only gene therapy treatment for severe hemophilia A,” Bienaimé said.

According to BioMarin, about 2,500 people in the U.S. are eligible for the treatment, and the company has tapped Orsini Specialty Pharmacy to distribute it.

Roctavian also was conditionally approved in the European Union for the same indication last year.

BioMarin is working directly with the German National Association of Statuary Health Insurance Funds to finalize a reimbursement deal for the therapy there. In the meantime, eligible patients in Germany can access Roctavian through “named patient” authorizations or previously secured “outcomes based agreements.”

The company also is in active talks with the single-payer public health insurance systems in Italy and France to secure reimbursement deals in those countries, which are expected to be finalized by the end of the year.

“For the remainder of 2023, we plan to build on the foundation of growth and profitability achieved in the first half of the year, … and treat the first Roctavian patients in the U.S. and Europe,” Bienaimé said.

BioMarin is conducting studies aiming to make Roctavian available to a wider number of patients, including research exploring strategies to effectively administer the therapy to people with anti-AAV5 antibodies or those with inhibitors.