Expert Council Resolution«Gene therapy for haemophilia B: a new reality»

Abstract

Summary. On 9 June 2025, an Expert Council convened in Moscow to discuss current challenges in the management of haemophilia B. The panel noted persistently low treatment adherence and suboptimal efficacy of existing therapeutic approaches. To address these concerns, the experts reviewed the potential role of Arvenacogene sanparvovec, the first gene therapy product developed in Russia for the treatment of haemophilia B. The discussion also covered the clinical profile of eligible candidates for such innovative treatment, as well as potential models for financing gene therapy within the healthcare system. The Council reached consensus that Arvenacogene sanparvovec shows considerable promise in enabling patients with haemophilia B to achieve subnormal haemostasis. Interim results from the Phase I/II ANB-002–1/SAFRAN trial demonstrated satisfactory efficacy and a favourable safety profile following a single administration at a specified dose. According to current data, the therapy is deemed suitable for adult patients (≥ 18 years) with haemophilia B and < 2% baseline factor (F) IX activity, who have no history of FIX inhibitors, no prior exposure to gene therapy, no active hepatitis B or C infection, and anti-AAV5 antibody titers below 1:5. The experts further recommended that, following regulatory approval, Arvenacogene sanparvovec be incorpo- rated into the national clinical guidelines for haemophilia. In order to ensure the successful integration of gene therapy into routine clinical practice, the Council advised implementing structured long-term follow-up to assess efficacy, safety, benefit-risk balance, and pharma- coeconomic outcomes. Additionally, the experts proposed the development of patient referral pathways, the establishment of centers of excellence for gene therapy, and the identification of sustainable funding mechanisms in collaboration with governmental health authorities.

For citation: Parovichnikova E.N., Ptushkin V.V., Zozulya N.I., Mamaev A.N., Davydkin I.L., Pospelova T.I., Mitina T.A., Konstantinova V.N., Baibulatova A.F., Bochkarev P.Yu., Dreval R.O., Teryan R.A. Expert Council Resolution «Gene therapy for haemophilia B: a new reality». Tromboz, gemostaz i reologiya. 2025;(3):87–91. (In Russ.).

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