ROCTAVIAN is First Gene Therapy for Hemophilia to Receive Approved Federal Price in Germany

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced an agreement with the German National Association of Statutory Health Insurance Funds (GKV-SV) on the reimbursement amount for ROCTAVIAN® (valoctocogene roxaparvovec-rvox) for people with severe hemophilia A. This is the first gene therapy for hemophilia to receive an agreed federal price in Germany. ROCTAVIAN brings value to individuals and the healthcare system in Germany, as a one-time, single-dose therapy delivered as an intravenous infusion over 3-4 hours at a gross price of €28,933.53 per vial. This vial price equates to approximately $900,000 per patient in net revenue to BioMarin at the current exchange rate, accounting for average patient weight and number of vials required for the one-time treatment, estimated reductions for customary rebates and discounts.

“We are pleased to have reached an agreement on the reimbursement amount for ROCTAVIAN with the GKV-SV, which is responsible for health insurance that covers approximately 90% of the German population,” said Jeff Ajer, executive vice president and chief commercial officer at BioMarin. “This important progress will facilitate access to ROCTAVIAN for eligible individuals among the roughly 2,000 adults with severe hemophilia A in Germany and marks a key step forward for the hemophilia community.”

BioMarin and GKV-SV have agreed on an outcome-based prospective cohort model for ROCTAVIAN. This will allow future reimbursement to be increased or decreased, based on real-world data collected from the German Haemophilia Registry of patients treated with ROCTAVIAN. This agreement, with a minimum term of 3 years, ensures the supply and reimbursement for eligible patients in Germany with ROCTAVIAN by the GKV-SV.

Two patients in Germany have now received ROCTAVIAN commercially, and 60 people are eligible for next steps based on companion diagnostic testing results for adeno-associated virus serotype 5 (AAV5) antibodies.

Additionally, final price negotiations with the Italian Medicines Agency are underway and expected to be formalized by year-end 2023.


Significant Progress with U.S. Payers

BioMarin continues to work with private and public payers in the U.S. to enable access, with the goal of ensuring that every eligible adult interested in ROCTAVIAN is able to receive treatment.

Numerous coverage policies that now include ROCTAVIAN have been published by leading U.S. payers. While medical exception processes provide a pathway to coverage, these policies representing millions of lives will help increase access to treatment.

The company continues to partner with hemophilia treatment centers (HTCs) across the country to educate them on how to administer ROCTAVIAN, and many HTCs have readiness plans before the end of this year.

It is estimated that there are approximately 6,500 adults living with severe hemophilia A in the U.S. BioMarin expects approximately 2,500 of those adults to be eligible to receive ROCTAVIAN at this time.


About Hemophilia A

Hemophilia A, also called factor VIII (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.



ROCTAVIAN is an adeno-associated virus vector-based gene therapy used for the treatment of adults with severe hemophilia A who do not have antibodies to adeno-associated virus serotype 5 (AAV5), which is determined by a blood test. The one-time infusion works by delivering a functional gene that is designed to enable the body to produce FVIII on its own, reducing the need for ongoing prophylaxis.

The European Commission (EC) granted conditional marketing authorization to ROCTAVIAN on August 24, 2022. The U.S. Food and Drug Administration (FDA) approved ROCTAVIAN on June 29, 2023.

About PackGene

PackGene is a CRO & CDMO technology company that specializes in packaging recombinant adeno-associated virus (rAAV) vectors. Since its establishment in 2014, PackGene has been a leader in the AAV vector CRO service field, providing tens of thousands of custom batches of AAV samples to customers in over 20 countries. PackGene offers a one-stop CMC solution for the early development, pre-clinical development, clinical trials, and drug approval of rAAV vector drugs for cell and gene therapy (CGT) companies that is fast, cost-effective, high-quality, and scalable. Additionally, the company provides compliant services for the GMP-scale production of AAVs and plasmids for pharmaceutical companies, utilizing five technology platforms, including the π-Alpha 293 cell AAV high-yield platform and the π-Omega plasmid high-yield platform. PackGene's mission is to make gene therapy affordable and accelerate the launch of innovative gene drugs. The company aims to simplify the challenging aspects of gene therapy development and industrialization processes and provide stable, efficient, and economical rAAV Fast Services to accelerate gene and cell therapy development efforts from discovery phase to commercialization.

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