German health insurance fund to reimburse Roctavian for hem A

BioMarin reaches deal aiming to improve patient access to gene therapy

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by Mary Chapman |

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Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association of Statutory Health Insurance Funds, known as GKV-SV.

The one-time intravenous or into-the-vein treatment is the first hemophilia gene therapy to have an agreed-upon federal price in Germany.

Roctavian’s gross price is now set at €28,933.53 (just less than $31,500) per vial, which comes out to approximately $900,000 in net revenue per patient to California-based BioMarin at the current exchange rate. The pricing accounts for average patient weight, the number of vials needed, and approximated reductions for customary discounts and rebates.

“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,” Jeff Ajer, BioMarin’s executive vice president and chief commercial officer, said in a company press release.

“This important progress will facilitate access to Roctavian for eligible individuals among the roughly 2,000 adults with severe hemophilia A in Germany,” Ajer added.

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In addition, an agreement between the two entities on an outcome-based prospective Roctavian cohort (group) model will permit future reimbursement to increase or decrease. The reimbursement level will depend upon real-world data from the German Haemophilia Registry of patients treated with the single-dose therapy.

The agreement, with terms of at least three years, ensures the supply of Roctavian and reimbursement for eligible patients in Germany by the GKV-SV.

To date, two patients in Germany have been commercially treated with the therapy, with the first recipient announced in August.

Another 60 individuals are eligible to advance toward treatment based on diagnostic test results for adeno-associated virus serotype 5 (AAV5) antibodies. AAV5 DetectCDx, a companion diagnostic test used to determine treatment eligibility, recently was cleared for use under tighter European Union rules.

[The new agreement] marks a key step forward for the hemophilia community.

BioMarin is seeking to increase access to Roctavian in the U.S. and in the rest of Europe. To that end, final price negotiations are underway with the Italian Medicines Agency, which are expected to be finalized by year’s end. Roctavian was given conditional approval in the European Union last year as a treatment for adults with severe hemophilia A.

Following the therapy’s approval by the U.S. Food and Drug Administration in June, it became available by the end of summer for most U.S. patients.  There are an estimated 6,500 adults living with severe hemophilia A in the country, and the company expects 2,500 of them to be eligible for Roctavian treatment.

BioMarin is continuing to work with private and public U.S. payers to enable access, and a number of coverage policies that now include the therapy have been published. Such policies, representing millions of patients, will help enhance treatment access, according to the company.

The company is continuing to educate U.S. hemophilia treatment centers on Roctavian administration; many are expected to have readiness plans established by the end of the year.

Roctavian is crafted to deliver a healthy copy of the F8 gene to patient cells. Hemophilia A results from mutations in that gene, causing a deficiency in a clotting protein — factor VIII — and putting those with the disorder at risk of experiencing excessive and prolonged bleeding.

By delivering a functional copy of the F8 gene to the liver, where clotting factors are produced, the treatment is designed to restore the production of the protein, reducing bleed risk and prospectively lowering or eliminating the necessity for standard routine preventive treatment. The delivery occurs via a virus known as AAV5, which was engineered to deposit the gene within cells rather than cause an infection.

According to Ajer, the new agreement “marks a key step forward for the hemophilia community.”