UK and Denmark Approve CSL's Hemgenix Gene Therapy for Haemophilia B with Novel Reimbursement Models
Key Insights
NICE reverses earlier rejection of Hemgenix in England, implementing a first-of-its-kind managed access programme through the Innovative Medicines Fund for haemophilia B (search) treatment.
Denmark becomes the first Nordic country to recommend reimbursement of Hemgenix, priced at DKK 21.5 million ($3.1 million), marking significant expansion in European access.
The one-time gene therapy, while carrying a £2.6 million price tag, offers potential long-term cost savings compared to traditional factor IX replacement therapies costing up to £200,000 annually.
CSL Behring has secured crucial reimbursement approvals for its groundbreaking haemophilia B (search) gene therapy Hemgenix in both England and Denmark, marking significant advancement in treatment accessibility for patients with severe or moderately severe disease.
In a notable policy reversal, the UK's National Institute for Health and Care Excellence (NICE) has approved Hemgenix coverage through NHS England (search), following initial rejection last year. The approval applies to adult patients with severe or moderately severe haemophilia B (search) who have no history of factor IX inhibitors.
Novel Payment Model and Access Programme
The approval comes with an innovative managed access programme under the Innovative Medicines Fund (IMF), representing the first instance of an advanced therapy medicinal product utilizing an outcomes-based payment model within the UK's voluntary rebate scheme. This arrangement allows for continued evidence gathering while providing immediate patient access.
The therapy's £2.6 million ($3.3 million) list price in the UK is offset by the substantial long-term savings potential, considering that current factor IX replacement therapies cost approximately £200,000 per patient annually, not including additional healthcare delivery costs and bleeding episode management.
Nordic Market Entry and Clinical Impact
Simultaneously, Denmark has emerged as the first Nordic nation to recommend Hemgenix reimbursement, setting the stage for a commercial agreement with Amgros (search), the Danish procurement authority. The therapy is priced at DKK 21.5 million ($3.1 million) in the Danish market.
Hemgenix operates as a single-infusion treatment designed to correct factor IX deficiency, potentially eliminating or significantly reducing the need for regular factor IX replacement therapy. Clinical trial data has shown that some patients have maintained independence from factor IX medications for several years post-treatment.
Patient Benefits and Healthcare Impact
"This is a major step forward in the treatment options for people living with severe haemophilia B (search)," stated Clive Smith, chair of the Haemophilia Society (search). "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."
The therapy's approval addresses significant unmet needs in haemophilia B (search) treatment, offering patients the potential for long-term relief from regular infusions while providing healthcare systems with a novel approach to managing treatment costs through innovative payment models.
Regulatory Progress Across Europe
The therapy's acceptance in the UK and Denmark follows its recent authorization under France's 'direct access' programme, demonstrating growing European recognition of its therapeutic value. These approvals reflect an evolving approach to evaluating and implementing advanced therapeutic options, particularly in the realm of gene therapy.