New Gene Therapy for Haemophilia

Gene therapy Hemgenix (etranacogene dezaparvovec) will be available on the NHS as a treatment option for adults with moderately severe or severe haemophilia B. The drug has been recommended for NHS use by the National Institute of Health and Care Excellence (NICE) with managed access, while further clinical evidence is gathered.

Sir Stephen Powis, NHS National Medical Director, said: “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.

“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.

“This promising drug is the latest in a series of pioneering gene therapies secured for NHS patients at an affordable price and becomes the first drug to be made available in our Innovative Medicines Fund to provide early access for patients while further data is collected on its long-term benefits.”

Haemophilia is a rare condition that affects the blood’s ability to clot. It’s usually inherited. Most people who have it are male.

Normally, when you cut yourself, substances in your blood known as clotting factors mix with blood cells called platelets to make your blood sticky and form a clot. This makes the bleeding stop eventually.

People with haemophilia do not have as many clotting factors as they should have in their blood. This means they bleed for longer than usual.

Symptoms of Haemophilia

The symptoms of haemophilia can be mild to severe, depending on the level of clotting factors you have.

The main symptom is bleeding that does not stop.

People with haemophilia may have:

  • Nosebleeds that take a long time to stop
  • Bleeding from wounds that lasts a long time
  • Bleeding gums
  • Skin that bruises easily
  • Pain and stiffness around joints, such as elbows, because of bleeding inside the body (internal bleeding)

Read more about the symptoms of haemophilia.

Clinical trial evidence shows that the gene therapy etranacogene dezaparvovec reduces the number of bleeding episodes a person has each year.


Hemgenix becomes the first treatment to enter NHS England’s Innovative Medicines Fund (IMF) with managed access, following a commercial agreement between NHS England and CSL Behring, the drug’s manufacturer. The IMF was established to support patient access to pioneering non-cancer medicines, such as cell and gene therapies, at a responsible price. This new gene therapy today follows other gene therapies available on the NHS, including Zolgensma and Libmeldy.

Through the IMF, NHS England will immediately fund the treatment at eight specialist centres across England (in two in London, Oxford, Manchester, Leeds, Bristol, Birmingham and Cambridge)
the gene therapy is given by a one-off infusion which takes around 1-2 hours. Currently, treatment with Factor IX is repeated regularly and people with severe haemophilia B typically need infusions once or twice a week.

An estimated 2,000 people in the UK have haemophilia B and around 250 will be eligible for the new treatment in England

Patients with haemophilia B can speak to their healthcare professional to discuss eligibility and tolerance for treatment, which may take several months prior to administration.

See also: Getting Down to the Nitty Gritty – Head Lice Advice From the Experts


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