A new gene therapy for haemophilia B is to be made available for NHS patients after approval from the UK's National Institute for Health and Care Excellence (NICE).
Through the NHS England's Innovative Medicines Fund, the gene therapy, Hemgenix (etranacogene dezaparvovec), will immediately be available at eight specialist centres across England (two in London and one in each of Oxford, Manchester, Leeds, Bristol, Birmingham, and Cambridge). Hemgenix is used to treat adults with moderately severe and severe haemophilia B, a genetic condition caused by the partial or complete loss of factor IX, a protein essential to produce blood clots.
'This transformative gene therapy is the first of its kind for haemophilia B patients on the NHS and has the potential to significantly improve the lives of hundreds of people by helping to reduce symptoms such as painful bleeds,' Professor Sir Stephen Powis, NHS national medical director, said.
Approximately 2000 people in the UK have haemophilia B and NICE predicts that around 250 of these patients will be eligible for the gene therapy in England. The treatment will be available to adults with moderately severe or severe haemophilia B who have no history or current factor IX inhibitors, which are proteins made by the body's natural defences.
Currently treatments for severe haemophilia involve factor IX infusions once or twice a week, which increase factor IX levels temporarily. Yearly, these treatments can cost the NHS £150,000-£200,000 per patient. Contrastingly, Hemgenix is administered as a single infusion over one to two hours.
'At its most effective, gene therapy has the potential to transform lives by eliminating painful bleeds and removing the need for regular, invasive, treatment,' Clive Smith, chair of the Haemophilia Society, said.
Evidence indicates that Hemgenix reduces the number of bleeding episodes a person has a year. Yet, the treatment was initially rejected by NICE, due to uncertainties in the long-term clinical evidence and its cost-effectiveness (see BioNews 1202).
NICE has shifted its recommendation after the NHS England agreed a commercial deal with its manufacturer, CSL Behring, under an outcomes-based payment model. This means that the £2.6 million treatment will have managed access, which is a programme used to allow patients with serious or life-threatening conditions to access investigational treatments that are not yet available in order for NICE to collect further clinical data to confirm effectiveness.
Elliott Mason who has severe haemophilia B and took part in the gene therapy trials five years ago, told Royal Free London: 'My results were fantastic – it's been amazing for me. I get tested every six months and so far I haven't needed any further treatment'.
NICE draft guidance is still open to comment until 11 July before the NICE committee reassesses its initial recommendation and expect to publish their final decision on 24 July.
Sources and References
-
Haemophilia gene therapy now available to NHS patients
-
Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B [ID3812]
-
Responding to new final draft guidance from NICE for a new gene therapy for haemophilia B
-
I have £2.6m blood and it has 'cured' my haemophilia
-
NHS £2.6m blood infusion transforms life for haemophilia B patients
-
Transformational gene therapy approved for haemophilia B
Leave a Reply
You must be logged in to post a comment.