MHRA Grants Conditional Marketing Authorisation in Great Britain to CSL’s HEMGENIX® (etranacogene dezaparvovec) as First Gene Therapy for Haemophilia B

pharmafile | March 27, 2023 | News story | Business Services  

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

HAYWARDS HEATH, ENGLAND – March 27th 2023 – Global biotech leader CSL, has today announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional* marketing authorisation to HEMGENIX® (etranacogene dezaparvovec), as the first and only single infusion gene therapy for the treatment of severe and moderately severe haemophilia B (congenital FIX deficiency) in adults without a history of Factor IX (FIX) inhibitors in Great Britain (England, Scotland and Wales).

The MHRA decision follows conditional marketing authorisation from the European Commission (including Northern Ireland and the Republic of Ireland) and is based on results from the ongoing Phase III, open label, single-dose, single arm, multi-centre HOPE-B trial of 54 participants, the largest gene therapy trial in haemophilia B to date.1 Data from the trial show that haemophilia B patients treated with a single infusion of etranacogene dezaparvovec demonstrated significant increases in mean FIX activity levels of 36.9% at 18-months, that were sustained at 36.7% at 24-months post-treatment, compared to the six-month lead in period. 1 This led to an adjusted annualised bleed rate (ABR) reduction of 64% during months 7-18 (primary end-point) and 7-24 of the study compared to the same lead-in period pre-infusion (mean 4.18 vs 1.51; p=0.0002). 1Following infusion of etranacogene dezaparvovec, 96% of patients (52/54) discontinued routine FIX prophylaxis. 1 . Of the adverse events reported 24 months post-infusion, 424 (76%) were mild, 115 (21%) were moderate and 18 (3%) were severe. No inhibitors to FIX were reported.

“The positive decision from the MHRA is the essence of great science delivering a medicine that we believe could potentially change the treatment paradigm for both people living with haemophilia B and the healthcare professionals who treat them,” said Dr. Bill Mezzanotte, Head of Research and Development and Chief Medical Officer, CSL. “This asset, and our partnership with uniQure, underscore CSL’s promise to pursue, develop and deliver new treatments, particularly in disease states we know well like haemophilia B.”

The multi-year clinical development of etranacogene dezaparvovec was led by uniQure (Nasdaq: QURE) and sponsorship of the clinical trials transitioned to CSL after it licensed global rights to commercialise the treatment.

Haemophilia is a rare genetic bleeding disorder, in which a genetic mutation causes coagulant FIX, an important clotting protein, to be deficient or missing. The disease is characterised by bleeding episodes, pain, and long-term complications such as joint damage.2,3,4 Almost 2,000 people live with haemophilia B in the UK,5 those with severe disease currently require lifelong treatment of intravenous FIX (FIX prophylaxis) to maintain sufficient levels, which can have a significant impact to their quality of life. 6,7,8,9

Stephen Allan, Country Manager, CSL Behring UK and Ireland, said: “Today’s decision represents an important milestone for a potential new therapy option for people living with haemophilia B in the UK. We are now committed to working collaboratively with the National Institute for Health and Care Excellence to provide access to this innovative treatment for eligible patients across the UK.”

*Conditional marketing authorization is intended for medical products that fulfill an unmet medical need where the benefit of immediate availability of the medicine outweighs the risk inherent in the fact that additional data are still required.


1 Pipe SL, F; Recht, M; Key, N; Lattimore et al. W. 2141 Adults with Severe or Moderately Severe Hemophilia B Receiving Etranacogene Dezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up. 64th ASH Annual Meeting and Exposition; 10 December 2022, 2022; New Orleans, Louisiana.

2 Srivastava A et al. Haemophilia 2020;26(Suppl 6):1–158

3 Mannucci PM. Haematolgica 2020;105:545–553

4 Marchesini E, et al. Biologics. 2021; 15:221-235

5 United Kingdom Haemophilia Centres Doctors Organisation. UKHCDO Annual Report 2022 & Bleeding Disorder Statistics for the Financial Year 2021/22. content/uploads/2022/12/UKHCDO-Annual-Report-2022-2021-22-Data.pdf [Accessed February 2023]

6 Witkop M et al. Am J Hematol 2015;90(Suppl 2):S3–10;

7 Schrijvers LH et al. Br J Haematol 2016;174:454–460;

8 Thornburg CD et al. Patient Prefer Adherence 2017;11:1677–1686;

9 Forsyth AL et al. Patient Prefer Adherence 2015;9:1549–1560

10 Simioni P, Tormene D, Tognin G, et al. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med 2009;361(17):1671-5. (In eng). DOI: 10.1056/NEJMoa0904377.

11 Spronck EA, Liu YP, Lubelski J, et al. Enhanced Factor IX Activity following Administration of AAV5- R338L “Padua” Factor IX versus AAV5 WT Human Factor IX in NHPs. Mol Ther Methods Clin Dev 2019;15:221-231. (In eng). DOI: 10.1016/j.omtm.2019.09.005

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