The first haemophilia B patient in Germany was treated with the gene therapy Hemgenix® (etranacogene dezaparvovec) at Hannover Medical School.
Hemgenix is the first one-time gene therapy approved in Europe for the treatment of adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of Factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults without a history of Factor IX inhibitors.1
“Each advancement in haemophilia treatment, particularly through innovative therapies like gene therapy, brings us closer to a better and more carefree life,” explains Christian Schepperle, Managing Director of the Interessengemeinschaft Hämophiler e.V. (IGH) in Germany. “Our objectives in haemophilia treatment align closely with economic considerations. 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 is now available to patients in Germany under an innovative, success-based reimbursement model being implemented for the first time in the country.
Hemgenix was granted conditional marketing authorisation by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the US Food and Drug Administration (FDA) in November 2022. It has also received regulatory approval in Canada, the UK, Switzerland, Australia, Saudi Arabia, Taiwan, South Korea and Hong Kong. Following these approvals, CSL Behring is working with relevant stakeholders to ensure eligible patients have access to Hemgenix across Europe and beyond, building on milestone access decisions in Germany, Denmark, Switzerland, Spain, the UK (including Scotland), and Austria.2-7 Patients in France, Denmark, Austria, Spain and the UK have already experienced treatment with Hemgenix.
