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‘Transformative’ haemophilia gene therapy now available to NHS patients

NICE has approved the gene therapy for treatment, which could help people living with haemophilia live ‘full and independent lives’

The National Institute for Health and Care Excellence (NICE) has approved a gene therapy for treatment of haemophilia B on the NHS. The £2.6 million gene therapy could reduce the need for regular shots in people with the rare blood disorder.

The new gene therapy, Hemgenix (etranacogene dezaparvovec) will be the first treatment to enter NHS England’s Innovative Medicines Fund (IMF) following more than 14 years of research into gene therapy led by Professor Ted Tuddenham and Professor Amit Nathwani at the Royal Free Hospital in London.

Clive Smith, chair of the Haemophilia Society said this move by NICE was a ‘major milestone’ for the haemophilia community.

He said: ‘At its most effective, gene therapy has the potential to transform lives by eliminating painful bleeds and removing the need for regular, invasive, treatment. This is another important step towards our goal that everyone living with an inherited bleeding disorder has access to treatment which allows them to lead a full and independent life.’

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There are currently about 2,000 people in the UK who live with haemophilia B. It is a lifelong, inherited bleeding disorder where factor IX (nine), one of the clotting factor proteins important for blood clotting, is either partly or completely missing.

People with the condition can experience severe and painful bleeding after minor injuries and have to get regular factor IX injections to help their bodies heal. In the research trials at Royal Free Hospital, the gene therapy helped many patients stay off injections for years.

Stephen Powis, national medical director for NHS England said: ‘This promising drug is the latest in a series of pioneering gene therapies secured for NHS patients at an affordable price. The transformative therapy has the potential to significantly improve the lives of hundreds of people.’