In the UK, the National Institute for Health and Care Excellence (NICE) recommended the gene therapy for reimbursement by the National Health Service (NHS) via a managed access agreement under the Innovative Medicines Fund (IMF), which is designed to speed up the access of patients to new treatments.

CSL Behring, the developer of Hemgenix, hailed this as the first arrangement of its kind with a gene therapy and the first advanced therapy to use an outcome-based payment model, which was set up by the UK health system to improve patient health while keeping costs low.

“This is a major step forward in the treatment options for people living with severe haemophilia B,” said Clive Smith, Chair of the Haemophilia Society, in a public statement​. “It has the potential to significantly improve the quality of life of those who are eligible for such treatment. Gene therapy provides an opportunity for people to effectively eliminate painful bleeds, thereby improving joint health and allowing people to lead a full life, unrestricted by frequent infusions and trips to hospital.”

Also recently, Denmark became the first Nordic country to set up the reimbursement of Hemgenix, with the company now working to expand the treatment’s reach across Europe.

Hemophilia B​ is a rare genetic disorder where a patient lacks healthy copies of factor IX (FIX), a protein that is essential for clotting the blood. As a result, patients bleed for longer than healthy people and require intravenous infusions of FIX replacement therapy. Those with severe forms of the condition can require prophylactic treatment with FIX to prevent bleeds.