Welcoming a new era of innovative treatment for haemophilia patients
With therapies that can change a patients’ DNA, what once seemed like science fiction is now science fact.
Since 2021, NHS England has commissioned three gene therapies for the treatment of specific rare genetic conditions, including onasemnogene abeparvovec (Zolgensma®) for spinal muscular atrophy (SMA), atidarsagene autotemcel (Libmeldy®) for metachromatic leukodystrophy and eladocagene exuparvovec (Upstaza®) for aromatic L-amino acid decarboxylase (AADC) deficiency. Alongside groundbreaking CAR T therapies for five different adult and childhood cancer indications, which use the body’s own immune system to attack cancer cells, the portfolio of NHS medicines is increasingly made up of what is collectively known as Advanced Therapy Medicinal Products (ATMPs).
These treatments have heralded a transformation in care for patients affected by these conditions, and the next group of patients to benefit from the arrival of an ATMP are those with haemophilia.
Haemophilia is a lifelong, inherited bleeding disorder that causes people to bleed for longer than normal, due to a problem with blood clotting. There are different types of haemophilia, based on the genetics of the condition – the different types have the same symptoms, but different treatments.
Last month, a new gene therapy called etranacogene dezaparvovec (Hemgenix®), the first treatment funded by NHS England via the Innovative Medicines Fund (IMF), was made available for patients with severe or moderately severe haemophilia B. Patients with haemophilia B are either partly or completely missing clotting factor IX, and may have issues with spontaneous internal bleeding without injury, as well as traumatic bleeding when injured.
If successful, the one-off gene therapy could eliminate the need for regular injections of factor IX and prevent patients from having to experience painful bleeds. The therapy works by introducing a working version of the factor IX gene into the body, enabling the liver to start making its own factor IX, and therefore allowing beneficial blood clots to form when needed.
The National Institute for Health and Care Excellence (NICE) estimates that there are around 250 patients that will meet the eligibility requirements for treatment in England.
The new treatment is available via eight new haemophilia ATMP hubs, balancing the required expertise of the provider, geographical access and the ability to offer patients such highly specialised treatment. It is expected that further ATMPs could be made available for haemophilia types A and B in the near future, and so the new ATMP hubs will be set up to provide the expert care to successfully deliver these treatments.
The eight new haemophilia ATMP hubs, covering every NHS region in England, are located at:
- Guys and St Thomas’ NHS Foundation Trust
- Royal Free London NHS Foundation Trust
- Manchester University Foundation Trust
- Oxford University Hospitals NHS Foundation Trust
- Leeds Teaching Hospitals NHS Trust
- University Hospitals Bristol and Weston NHS Foundation Trust
- University Hospitals Birmingham NHS Foundation Trust
- Cambridge University Hospitals NHS Foundation Trust
Etranacogene dezaparvovec is the first treatment to enter NHS England’s IMF, following a commercial deal being reached between NHS England and the drug’s manufacturer, CSL Behring. Through the IMF, which was set up to support patient access to pioneering medicines, NHS England is immediately funding the treatment for patients. The Cancer Drugs Fund (CDF) has provided immediate funding for cancer treatments since 2011, and the IMF offers an equivalent route for non-cancer medicines.
Typically, there is a 90-day period between final guidance from NICE and funding, but the IMF allows for immediate funding for commissioned treatments, enabling clinicians to start patients on the treatment pathway right away.
It’s fantastic news that patients with this severe form of haemophilia will be able to benefit from a transformative gene therapy, which has the potential to significantly improve their quality of life. This deal also highlights the agile nature of the IMF, allowing access to pioneering treatments even faster.
Ben Doak
Ben Doak is the Head of Innovative Treatments at NHS England and has worked for the NHS since 2005 in a number of roles supporting evidence-based patient care. With his team, Ben is responsible for ensuring that services are in place so that patients can benefit from licensed Advanced Therapy Medicinal Products (ATMPs) recommended by NICE (the National Institute of Health and Care Excellence) in the NHS in England.
Since commencing his current role in 2020, Ben has overseen the expansion of CAR-T services in England, as well supporting access to ATMPs such as Zolgensma for spinal muscular atrophy, Libmeldy for metachromatic leukodystrophy and Upstaza for AADC deficiency. He maintains an active interest in horizon scanning of ATMPs and other innovative treatments.