A New Era of Treatment for Haemophilia Patients

With therapies that can change a patient’s DNA, the NHS is ushering in a new era of treatment that can turn science fiction into fact

CREDIT: This is an edited version of an article that originally appeared on NHS England

Since 2021, NHS England has commissioned three gene therapies for the treatment of specific rare genetic conditions. In addition to the groundbreaking CAR T therapies for five different adult and childhood cancers, which harness the body’s immune system to target cancer cells, the NHS medicine portfolio is increasingly incorporating Advanced Therapy Medicinal Products (ATMPs). The next group of patients set to benefit from ATMPs are those with haemophilia.

Haemophilia is a lifelong, inherited bleeding disorder that impairs blood clotting, causing prolonged bleeding. Recently, a new gene therapy called etranacogene dezaparvovec (Hemgenix®) has become available for patients with severe or moderately severe haemophilia B. Individuals with haemophilia B may experience spontaneous internal bleeding without injury, as well as excessive bleeding from injuries.

If successful, this one-time gene therapy could prevent patients from experiencing painful bleeds. The therapy functions by introducing a functional version of the factor IX gene into the body, enabling the liver to produce its own factor IX, which facilitates the formation of beneficial blood clots. According to the National Institute for Health and Care Excellence (NICE), approximately 250 patients in England will meet the eligibility criteria for this treatment.

It’s wonderful news that patients with this severe form of haemophilia will now have access to transformative gene therapy, which has the potential to significantly enhance their quality of life. The new treatment is now available through eight haemophilia ATMP hubs, designed to balance the provider’s expertise, geographical access, and the ability to deliver highly specialised care. These hubs are expected to facilitate the availability of additional ATMPs for haemophilia types A and B in the near future, ensuring expert care for the successful administration of these advanced therapies.