BioMarin secures hemophilia gene therapy coverage in Germany

Dive Brief:

• BioMarin Pharmaceutical has reached an agreement with German authorities on a price for Roctavian, more than one year after the gene therapy for hemophilia won European approval.
• The deal will provide about $900,000 in revenue to BioMarin per patient, the company said Tuesday. That’s well below the $2.9 million list price set in the U.S., which BioMarin executives previously said would translate to about $1.9 million in net revenue for the company. It’s also below the $1.5 million target BioMarin originally set for Europe.
• Still, BioMarin may be able to charge more in coming years. The agreement includes an outcome-based aspect, which allows future reimbursement to be raised or lowered depending on how well patients fare after treatment. Roctavian is a one-time infusion given over a period of three to four hours and has been shown to have lasting effects treating bleeding caused by hemophilia A.

Dive Insight:

Though BioMarin came away with a lower price than it might have hoped, the agreement with the German National Association of Statutory Health Insurance Funds is a significant milestone for the company, analysts said. The association handles the health insurance of about 90% of the German population and has proven to be a tough negotiator on gene therapy pricing.

“Securing reimbursement is a good step forward,” Stifel analyst Paul Matteis wrote in a note to clients. Matteis, who had modeled a net price of more than $1 million in the European Union, said the final figure is nonetheless “economically significant and sets Roctavian up to be a potentially meaningful product in Europe.”

The resolution of talks may also free up a significant bottleneck of patients in Germany waiting to receive the treatment. The company said 60 people are eligible for the next steps based on previous testing. Until now, just two patients in Germany have received Roctavian commercially.

BioMarin reiterated that it expects to have price negotiations concluded with the Italian Medicines Agency by the end of the year and said it’s seen “significant progress” with U.S. payers. The company estimates about 6,500 adults in the U.S. have severe hemophilia A and about 2,500 of them are eligible to receive Roctavian.

The actual price per patient in Germany will vary, in part because different numbers of vials are needed to treat patients of different weights. The gross price will be 28,933.53 euros, or about $32,000, per vial, BioMarin said.

Roctavian first won European approval in August 2022, with U.S. clearance following in June 2023. But negotiations with payers proved tricky, and far fewer patients have been given the therapy than originally expected. Earlier this month, the company lowered its revenue estimate for Roctavian to less than $10 million for this year and announced plans to bring on a new chief executive.