First Person Treated in Germany; Reimbursement Discussions on Track in France and Italy

In the U.S., ROCTAVIAN Now Commercially Available and Hemophilia Treatment Centers Have Begun Screening Eligible Individuals

SAN RAFAEL, Calif., Aug. 30, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that an individual in Germany with severe hemophilia A was treated with ROCTAVIAN® (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.

"Today represents an important milestone for the hemophilia community, and for patients and physicians around the world seeking access to ROCTAVIAN," said Jeff Ajer, executive vice president and chief commercial officer at BioMarin. "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."

Hemophilia treatment centers (HTCs) in Germany are testing people with hemophilia to determine treatment eligibility for the one-time gene therapy. Before beginning treatment, individuals are evaluated clinically and undergo testing. Currently, dozens of individuals are undergoing screening to determine eligibility for treatment with ROCTAVIAN. Final federal price negotiations in Germany are also ongoing. 

"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 PD Dr. med. Robert Klamroth, a treating physician and chief physician of 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."

Beyond Germany, the company's applications seeking price and reimbursement approvals and other launch preparation activities continue to progress in France and Italy. In Italy, ROCTAVIAN was awarded conditional innovation designation, which is expected to facilitate pricing and reimbursement.

U.S. Launch Activities Accelerating, including Eligibility Testing at HTCs

In June, the FDA approved ROCTAVIAN for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity <1 iu dl) without antibodies to adeno-associated virus serotype 5 (aav5) detected by an fda-approved test.< p>

ROCTAVIAN is now commercially available in the U.S. and HTCs have begun screening people with severe hemophilia A to determine eligibility. BioMarin has also seen an increasing inflow of patient consent forms and a number of executed or in-process warranty agreements that are expected to facilitate access and uptake of ROCTAVIAN at HTCs across the U.S.

About Hemophilia A

Hemophilia A, also called FVIII deficiency or classic hemophilia, is an X-linked genetic disorder caused by missing or defective FVIII, a clotting protein. Although it is passed down from parents to children, about one-third of cases are caused by a spontaneous mutation, a new mutation that was not inherited. Approximately 1 in 10,000 people have hemophilia A.

People living with hemophilia A lack sufficient functioning FVIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels