First patient with haemophilia B in the UK treated with CSL Behring’s gene therapy HEMGENIX® (etranacogene dezaparvovec) by the NHS

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The first patient with haemophilia B in the UK has been treated with HEMGENIX® (etranacogene dezaparvovec) by the NHS, paving the way for further eligible patients to access this one-time treatment option, and the possibility of a future free from routine prophylactic infusions.1,2

Etranacogene dezaparvovec is the first and only gene therapy for haemophilia B to receive a positive recommendation from the National Institute for Health and Care Excellence (NICE) through the Single Technology Appraisal (STA) process and the first medicine to be made available on the NHS through a managed access agreement via the Innovative Medicines Fund (IMF).3

This milestone underlines CSL Behring’s commitment to working closely with the NHS England selected gene therapy centres to ensure that eligible patients can access treatment.

 

▼ This medicine is subject to additional monitoring. This will allow quick identification of new safety information.

HAYWARDS HEATH, ENGLAND – 19th June 2025 – CSL Behring has announced that a patient with haemophilia B, from the North East of England, has been treated with HEMGENIX® (etranacogene dezaparvovec) at Guys & St Thomas’s NHS Foundation Trust, London, making them the first patient to receive this treatment on the NHS.

Etranacogene dezaparvovec is the first and only one-time gene therapy approved in the UK for adults with severe or moderately severe haemophilia B, without a history of Factor IX (FIX) inhibitors. 1,2 The milestone marks a significant step forward in potentially improving outcomes for patients with haemophilia B in the UK.

Dr Pu-Lin Luo, consultant haematologist at Guy’s and St Thomas’, said: “This is a big step forward in our ability to manage haemophilia B and could change the lives of some of our patients. It is also a testament to the advancement of cell and gene therapies in the UK and these are exciting times. Administering the first dose of etranacogene dezaparvovec outside of a clinical study was made possible by exceptional cross-centre collaboration between Guy’s and St Thomas’, where the patient was treated, and Newcastle Hospitals, their local haemophilia centre.”

Haemophilia B is an inherited bleeding disorder caused by a lack of FIX, an important blood-clotting protein. The disease is characterised by bleeding episodes, pain, and long-term complications such as joint damage.4,5,6 More than 2,000 people live with haemophilia B in the UK, a proportion of whom are eligible for consideration for gene therapy. 7 Those with severe disease currently require lifelong treatment of intravenous FIX (FIX prophylaxis) which can still leave patients vulnerable to breakthrough bleeds and pain in the days before their next infusion.5,8,9

Etranacogene dezaparvovec offers a one-time, one-hour, infusion treatment that addresses the underlying genetic cause of haemophilia B.10 It has demonstrated its potential as the first licensed gene therapy treatment option to significantly reduce the rate of annual bleeds with a single infusion, potentially reducing or eliminating the need for routine infusions, currently administered every 3-21 days.2

“Today marks an important milestone not just for this patient, but for all those living with haemophilia B in the UK,” said Kate Burt, Chief Executive Officer of the Haemophilia Society. “The current treatment of lifelong intravenous injections can place a significant burden on those living with haemophilia and has an impact on broader family, relationships and work. The availability of gene therapy for haemophilia B as a one-time infusion will allow those eligible for treatment to expand their horizons and live life to the full, free from the restrictions of regular injections.”

“This patient receiving etranacogene dezaparvovec is a testament to the collaborative efforts of the haemophilia community, NICE and NHS England to ensure that patients in the UK are able to access this one-time treatment option. Living with the persistent threat of spontaneous bleeds means that people with haemophilia are never free from thinking about their condition – etranacogene dezaparvovec has the potential to enable people living with haemophilia B to move towards a haemophilia-free mind.” Said Eduardo Cabas, General Manager, CSL Behring UK and Ireland. “CSL Behring remains committed to improving the lives of people with rare genetic bleeding disorders through innovative treatments, and we are excited to see the real-life impact of this therapy as further eligible patients across the country gain access to etranacogene dezaparvovec.”

Etranacogene dezaparvovec was recommended by NICE following the STA process for immediate reimbursement by NHS England through the first ever managed access agreement to be made under the IMF in June 2024.1,3

CSL Behring will continue to work closely and collaboratively with the selected gene therapy centres to ensure that further eligible patients across the country will be able to access this treatment. CSL Behring is also working with relevant stakeholders to ensure eligible patients have access to etranacogene dezaparvovec cross Europe, building on milestone access decisions in Germany, Denmark, Switzerland, Spain, the UK (including Scotland), Ireland and Austria. 1,11,12,13,14,15 Patients in France, Denmark and Austria have also already been treated with etranacogene dezaparvovec.

 

About etranacogene dezaparvovec

Etranacogene dezaparvovec is a gene therapy that reduces the rate of abnormal bleeding in eligible people with severe or moderately severe haemophilia B by enabling the body to continuously produce factor IX, the deficient protein in haemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV).10 The AAV5 vector carries the Padua gene variant of factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x more active than normal. 16 These genetic instructions remain in the target cells, but generally do not become a part of a person’s own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.17


About the HOPE-B Trial2,10

The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label, single-arm study to evaluate the safety and efficacy of etranacogene dezaparvovec. Fifty-four adult haemophilia B patients classified as having moderately severe to severe haemophilia B and requiring prophylactic factor IX replacement therapy were enrolled in a prospective, six-month or longer observational period during which time they continued to use their current standard of care therapy to establish a baseline Annual Bleeding Rate (ABR). After at least the six-month lead-in period, patients received a single intravenous administration of etranacogene dezaparvovec at the 2x1013 gc/kg dose. Patients were not excluded from the trial based on pre-existing neutralizing antibodies (NAbs) to AAV5.

A total of 54 patients received a single dose of etranacogene dezaparvovec in the pivotal trial, with 52 patients completing at least three years of follow-up. The primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement of stable factor IX expression (months 7 to 18) compared with the six-month lead-in period. For this endpoint, ABR was measured from month seven to month 18 after infusion, ensuring the observation period represented a steady-state factor IX transgene expression. Secondary endpoints included assessment of factor IX activity.

Of the adverse events reported 36 months post-infusion, 541 (76%) were mild, 137 (19%) were moderate and 31 (4%) were severe. No serious treatment-related adverse reactions were reported. One death (previously reported) resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65 weeks following dosing was considered unrelated to treatment by investigators and the company sponsor. A serious adverse event of hepatocellular carcinoma was determined to be unrelated to treatment with etranacogene dezaparvovec by independent molecular tumour characterisation and vector integration analysis. No inhibitors to factor IX were reported.

Long-term three-year data presented at the 65th American Society of Haematology (ASH) 2023 Annual Meeting and Exposition continue to reinforce the potential long-lasting efficacy and safety of etranacogene dezaparvovec and the ongoing possible benefit this treatment may offer people living with haemophilia B.

 

About CSL


CSL Limited is a global biotechnology company with a dynamic portfolio of potentially lifesaving medicines, including those that treat haemophilia and immune deficiencies, vaccines to prevent influenza, and therapies in iron deficiency and nephrology. Since our start in 1916, we have been driven by our promise to save lives using the latest technologies. Today, CSL – including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor – provides lifesaving products to patients in more than 100 countries and employs 32,000 people. Our unique combination of commercial strength, R&D focus and operational excellence enables us to identify, develop and deliver innovations so our patients can live life to the fullest.

 

Media contacts:

For UK and Europe enquiries:

Carol Chan
Senior Business Communications Partner
CSL Behring
Mobile: +852 6019 2690
Email: carol.chan@cslbehring.com

Joshua Vine-Lott
Account Director
Aurora Healthcare Communications
Mobile: +44 7969 109 622
Email: Joshua.vine-lott@auroracomms.com

 

References
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  1. National Institute for Health and Care Excellence. Technology Appraisal Guidance Etranacogene dezaparvovec for treating moderately severe or severe haemophilia B
  2. Pipe S.W., et al. Blood. 2023;142(Supplement 1):1055
  3. NHS England. News. Responding to new final draft guidance from NICE for a new gene therapy for haemophilia B. Available at: https://www.england.nhs.uk/2024/06/responding-to-new-final-draft- guidance-from-nice-for-a-new-gene-therapy-for-haemophilia-b/ [Last accessed June 2025]
  4. Srivastava A, et al. Haemophilia 2020;26(Suppl 6):1–158
  5. Mannucci PM. Haematolgica 2020;105:545–553
  6. Marchesini E, et al. Biologics. 2021; 15:221-235
  7. United Kingdom Haemophilia Centres Doctors Organisation. UKHCDO Annual Report 2023 & Bleeding Disorder Statistics for the Financial Year 2023/2024. https://www.ukhcdo.org/wp- content/uploads/2024/12/UKHCDO-Annual-Report-2024-2023-24-Data.pdf[Last accessed: June 2025]
  8. Schrijvers L.H., et al. Br J Haematol 2016;174:454–460;
  9. Thornburg C.D., et al. Patient Prefer Adherence 2017;11:1677–1686;
  10. Pipe S. W. et al. 2023;388(8), 706–718.
  11. CSL Bering. CSL Behring Signs First Commercial Agreement in Austria to Fund Haemophilia B Gene Therapy HEMGENIX®. Available at: https://www.cslbehring.de/en-us/news/2024/pm-hemgenix-agreement-austria [Last accessed June 2025]
  12. Medicinrådet. The Medical Council recommends the gene therapy Hemgenix following a new effect-based price agreement. Available at: https://medicinraadet.dk/nyheder/2024/medicinradet-anbefaler-genterapien-hemgenix-efter-ny-effektbaseret-prisaftale. [Last accessed June 2025].
  13. Scottish Medicines Consortium. Etranacogene dezaparvovec (Hemgenix). Available at: https://scottishmedicines.org.uk/medicines-advice/etranacogene-dezaparvovec-hemgenix-full-smc2649. [Last accessed June 2025].
  14. Ministerio De Sanidad. Puntos destacados de la reunión de la Comisión Interministerial de Precios de los Medicamentos 26 de septiembre de 2024. Available at: https://www.sanidad.gob.es/areas/farmacia/precios/comisionInteministerial/acuerdosNotasInformativas/docs/NOTAINFORMATIVACIPM_SEPTIEMBRE2024.pdf [Last Accessed: June 2025].
  15. Swiss Medic. HEMGENIX. Switzerland Authorisation. Available at: https://www.swissmedic.ch/swissmedic/en/home/about-us/publications/public-summary-swiss-par/public-summary-swiss-par-hemgenix.html [Last Accessed: June 2025]
  16. Spronck EA et al. 2019;15:221-231.
  17. Thornburg CD. Ther Adv in Rare Dis. 2021;2;1-14

 

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