HEMGENIX is the first one-time gene therapy to be 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.

A historic moment

“The first patients in Denmark to receive HEMGENIX mark a historic moment. Seventy-two years ago, it was discovered that inherited haemophilia B is caused by a genetic defect that deprives the liver of the capacity to produce enough functional Factor IX. Today, treating haemophilia B by restoring this capacity to the liver has become part of clinical practice in Denmark. This means that recurring factor-concentrate injections, repeated numerous times, can be replaced by a single infusion delivering the gene,” said Fredrik Sjöö, Head of Medical Affairs, Nordic Region, CSL Behring. “This is what scientific progress in medicine is intended to achieve: improving the life of patients, in particular those with rare diseases, by providing innovative therapeutic options.”

This makes Denmark the first Nordic and European country to adopt a performance-based model, where costs are incurred only as long as the gene therapy proves effective over the agreed long-term period.

Patients in Denmark can access HEMGENIX through an innovative outcome-based agreement with Amgros, finalized in October 2024. This makes Denmark the first Nordic and European country to adopt a performance-based model, where costs are incurred only as long as the gene therapy proves effective over the agreed long-term period.

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HEMGENIX 

HEMGENIX was granted conditional marketing authorization by the European Commission (EC) for the European Union and European Economic Area in February 2023, following approval from the U.S. Food and Drug Administration (FDA) in November 2022. It has also been approved by Health Canada, the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland’s Swissmedic, and Australia’s Therapeutic Goods Administration (TGA).

Following these approvals, CSL Behring is working with relevant stakeholders to continue making HEMGENIX available across Europe, building on milestone access decisions in Spain, Denmark, the UK (including Scotland), and Austria, so that more patients are able to receive the treatment, the company states.