Patient in Germany gets hemophilia B gene therapy Hemgenix
Public healthcare reimbursement tied to efficacy
The first hemophilia B patient in Germany has received the gene therapy Hemgenix (etranacogene dezaparvovec), according to CSL Behring, the therapy’s developer.
“The treatment of the first patient in Germany with Hemgenix marks a milestone in hemophilia B therapy,” Christian Wieszner, managing director of CSL Behring Germany, said in a company press release. “Treatments such as gene therapy have the potential to enable people living with haemophilia B to move towards a haemophilia-free mind.”
The cost of the one-time gene therapy will be covered for eligible patients in Germany through the country’s public healthcare system. Under the terms of an agreement between CSL Behring and GKV-Spitzenverband, the German National Association of Statutory Health Insurance Funds, reimbursement for Hemgenix will be tied to treatment efficacy, meaning the developer may offer refunds or discounts if the gene therapy doesn’t achieve certain clinical outcomes.
The agreement “addresses critical challenges such as the long-term efficacy of this one-time therapy and ensures that reimbursement is tied to the individual treatment success of each patient,” Wieszner said. “Additionally, it guarantees that the treatment costs for Hemgenix remain cost-neutral for statutory health insurance.”
Similar agreements have been reached in other European countries. The company said it’s “building on milestone access decisions” in Germany, Denmark, Switzerland, Spain, Austria, and the U.K. Eligible patients in France, Denmark, Austria, Spain, and the U.K. have been treated with Hemgenix, according to CSL Behring.
One-time infusion aims to simplify treatment
Hemophilia B is caused by mutations in the F9 gene that lead to a deficiency in factor IX, or FIX, a blood clotting protein, resulting in excessive or spontaneous bleeding episodes.
Hemgenix is designed to provide a working version of the F9 gene to hemophilia B patients via a one-time infusion into the bloodstream. It’s expected to prevent abnormal bleeding and eliminate or reduce the need for routine, preventive hemophilia therapies.
“Each advancement in hemophilia treatment, particularly through innovative therapies like gene therapy, brings us closer to a better and more carefree life,” said Christian Schepperle, managing director of the Interessengemeinschaft Hämophiler e.V. in Germany, a nationwide association that represents the interests of people with congenital bleeding disorders.
“Our objectives in hemophilia treatment align closely with economic considerations,” Schepperle said. “Advanced therapies are not only medically essential but also make socio-economic sense by helping to limit long-term healthcare costs — thus making us more economically and geopolitically independent.”
Hemgenix has been approved for hemophilia B in several countries, including the U.S., Canada, the U.K., Switzerland, Saudi Arabia, Taiwan, South Korea, Australia, and Hong Kong. In the European Union, it’s conditionally approved for adults with severe or moderately severe hemophilia B who do not have inhibitors, or neutralizing antibodies against FIX.
The approvals were supported by data from HOPE-B (NCT03569891), a Phase 3 trial that evaluated the safety and efficacy of Hemgenix in men with severe or moderately severe hemophilia B.
Four-year HOPE-B data, reported in February, showed that bleeding rates remain low, with sustained near-normal FIX activity. Most patients were able to discontinue routine preventive therapies, and no adverse events related to Hemgenix were reported.
In a study published last month on the long-term results of a Phase 1 (NCT00979238) clinical trial, the benefits of Hemgenix were sustained for up to 13 years in 10 men with severe hemophilia B, with no new safety concerns identified.
“CSL Behring is dedicated to providing opportunities for people with rare genetic bleeding disorders to better manage the impact of their condition on their daily lives,” Wieszner said.
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