CSL Behring’s HEMGENIX® Granted First-Ever Direct Access in France for Haemophilia B Gene Therapy

MARBURG, GERMANY – December 13, 2023 – Global biotechnology leader CSL Behring (ASX: CSL) has been granted Direct Access for HEMGENIX^® (etranacogene dezaparvovec) by the French Ministry of Health, marking the first treatment to be authorised by this innovative process in France.[i] HEMGENIX^® is the first and only one-time gene therapy for the treatment of severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.[ii] Through Direct Access, appropriate people living with haemophilia B will now be able to access HEMGENIX^®.

Patients with haemophilia B currently require lifelong treatment with intravenous Factor IX infusions, which can have a significant impact on quality of life and wellbeing.^[iii] According to the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA), there is an “unmet medical need for new therapeutic approaches that might free patients from the burden of frequent infusions, or episodically at the time of a bleeding event.”^[iv]

Following European marketing authorisation for HEMGENIX^® in February 2023, CSL Behring submitted a request for Direct Access to the Ministry of Health in early October 2023. This is the first time a medicine has been accepted via this exceptional access scheme in France and paves the way for other European countries to implement innovative access solutions.

“We are delighted and grateful for this historic decision from the Ministry of Health to grant patients Direct Access to HEMGENIX^® in France,” said Dr Lutz Bonacker, SVP and General Manager Commercial Operations Europe, CSL Behring. “It is hugely encouraging to see new pathways for access being explored, enabling eligible patients to receive gene therapy that could have a potentially transformative impact for them and their loved ones. This approval is an example of how effective collaboration and innovative thinking can deliver ground-breaking access solutions for patients across Europe, where there is still significant unmet patient need in haemophilia B.”

The Direct Access process is a two-year experimental measure, established by the French Social Security Financing Act (LFSS 2022).^[v] The programme allows treatments to be fully reimbursed by the French Health Insurance system for up to a year, following publication in the Official Journal.⁵

[i] Republique Française. Légifrance: Article 62 of Law No. 2021-1754. Available at: https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003 [Accessed December 2023].

[ii] European Medicines Agency. First Gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b. [Accessed December 2023].

[iii] Srivastava A et al. WFH Guidelines for the Management of Hemophilia, 3rd edition. Haemophilia 2020; 26(Suppl 6):1-158.

[iv] European Medicines Agency. First Gene therapy to treat haemophilia B. Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-haemophilia-b. [Accessed December 2023].

[v] Ministère de la Santé et de la Prévention. Dispositif d’accès direct pour certains produits de santé. Available at: https://sante.gouv.fr/soins-et-maladies/medicaments/professionnels-de-sante/autorisation-de-mise-sur-le-marche/article/dispositif-d-acces-direct-pour-certains-produits-de-sante#:~:text=L'objectif%20est%20de%20faire,compl%C3%A9ment%20de%20l'acc%C3%A8s%20pr%C3%A9coce [Accessed December 2023].