NHS Approves £2.6M Hemgenix Gene Therapy for Haemophilia B, Promising Decade-Long Relief
June 28, 2024CSL Behring's Hemgenix gene therapy for haemophilia B has received NHS approval in the UK.
NICE has granted coverage for adults with severe or moderately severe haemophilia B.
Denmark has recommended reimbursement for Hemgenix, the first Nordic country to do so.
The therapy costs £2.6 million per patient and is described as potentially 'transformative'.
Hemgenix aims to correct FIX deficiency and reduce the need for frequent replacement therapies.
Trial patients have reported staying off injections for years, with some feeling 'cured'.
The one-shot infusion therapy could last up to a decade.
The NHS will offer Hemgenix for free to around 250 eligible patients in England.
Around 260 out of 2,000 haemophilia B patients in the UK could benefit.
The Haemophilia Society has praised the potential of gene therapy to enhance quality of life.
Experts believe Hemgenix could save the NHS money by reducing the need for expensive treatments and surgeries.
The rollout marks a significant step in evaluating cell and gene therapies in the UK.
An outcomes-based payment model is being used for the first time.
NHS England is encouraging eligible patients to discuss Hemgenix with their healthcare professionals.
Summary based on 5 sources
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Sources
Forbes • Jun 27, 2024
U.K. Patients To Get $3.3 Million Hemophilia Gene Therapy For FreeBBC News • Jun 27, 2024
Haemophilia B: My £2.6m blood has 'cured' my haemophiliaDaily Mail • Jun 27, 2024
NHS watchdog approves 'world's most expensive drug' that costs £2.6million a dose... and it will...The Week • Jun 27, 2024
A 'transformative' gene therapy for haemophilia B