Hemgenix becomes the first treatment to enter NHS England’s Innovative Medicines Fund

In a ground-breaking development for the pharmaceutical industry and patients with haemophilia B, Hemgenix (etranacogene dezaparvovec) has become the first treatment to enter NHS England’s Innovative Medicines Fund (IMF) with managed access. This arrangement allows patients to receive promising treatments that might not be recommended otherwise due to uncertain clinical or cost-effectiveness evidence. This milestone follows a commercial agreement between NHS England and CSL Behring, the manufacturer of Hemgenix.

Hemgenix is a gene therapy that involves a one-time infusion lasting approximately 1-2 hours, offering a potentially curative treatment for haemophilia B. This therapy represents a significant advancement in managing this genetic disorder by reducing the frequency of bleeding episodes and decreasing the need for regular infusions of clotting factors, which are typically administered once or twice a week for severe cases.

The Innovative Medicines Fund is designed to fast-track promising treatments addressing unmet clinical needs, providing patients quicker access while data on their efficacy and safety is gathered. An estimated 2,000 people in the UK have haemophilia B, and about 250 of them will be eligible for this new treatment. Hemgenix’s inclusion in this fund marks a major step forward in adopting advanced therapies within the NHS framework, offering hope for individuals with rare and severe medical conditions.

Professor Sir Stephen Powis, NHS National Medical Director, commented, “It is a one-time therapy that could be truly life-changing for some, as it could help people avoid the need for regular hospital visits.”

Sources:

1. https://www.england.nhs.uk/2024/06/responding-to-new-final-draft-guidance-from-nice-for-a-new-gene-therapy-for-haemophilia-b/
2. https://www.precisionmedicineonline.com/regulatory-news-fda-approvals/csl-behrings-hemgenix-will-be-available-patients-england-under#:~:text=Hemgenix%20is%20the%20first%20treatment,at%20a%20%22responsible%20price.%22
3. https://haemophilia.org.uk/gene-therapy-haemophilia-b/