AIHTA - Publications - Search - Fidanacogene elaparvovec (BEQVEZ®) for the treatment of moderately severe to severe haemophilia B

Colicchia, A. and Fabian, D. and Grabenhofer, L. and Grössmann-Waniek, N. and Malíková, E. and Rothschedl, E. and Sehic, O. and Wild, C. and Wolf, S. and Zechmeister-Koss, I. (2025): Fidanacogene elaparvovec (BEQVEZ®) for the treatment of moderately severe to severe haemophilia B. Decision Support Document for the Austrian Appraisal Board 002.

[thumbnail of Appraisal_Board_Document_002.pdf] PDF - Sie müssen einen PDF-Viewer auf Ihrem PC installiert haben wie z. B. GSview, Xpdf oder Adobe Acrobat Reader
6MB
[thumbnail of Appendix] PDF (Appendix) - Sie müssen einen PDF-Viewer auf Ihrem PC installiert haben wie z. B. GSview, Xpdf oder Adobe Acrobat Reader
4MB
Abstract

Fidanacogene elaparvovec (BEQVEZ®) is the second gene therapy approved in Europe for the treatment of severe and moderate haemophilia B, which received conditional marketing authorisation from the European Commission on 24 July 2024. The medication, distributed by Pfizer, is classified as an "Advanced Therapy Medicinal Product" (ATMP). The approval covers the treatment of adults with no history of factor IX inhibitors and no detectable antibodies against AAVRh74var.

Fidanacogene elaparvovec demonstrated a 71% reduction in the annual bleeding rate compared to previous FIX prophylaxis therapy in a single-arm Phase 3 study (BENEGENE-2, n=45). The effect was maintained for up to 48 months. Side effects occurred in 84% of treated patients, with 16% experiencing serious reactions. The most common side effect was elevated aminotransferase levels (53%). Since the therapy is administered only once intravenously, surveyed patients express optimism about the reduced treatment burden and improved quality of life that may result from a simplified therapy form and treatment freedom. Given the existing uncertainties regarding long-term efficacy and potential side effects, it is recommended that patients receive the infusion in specialised centres with subsequent monitoring in local facilities. In addition, mandatory follow-up of all treated patients is considered necessary.

In Austria, 130 patients were reported by the Austrian Haemophilia Society (ÖHR) in 2024. Of these, 22.3% were affected by moderate haemophilia and 24.6% by severe haemophilia. According to clinical experts' assessment, nine patients would be eligible for the therapy with increasing uptake over three years (Year 1: 20%, Year 2: 30%, Year 3: 50%).

Item Type:DSD: Appraisal Board
Keywords:Haemophilia B, moderate to severe, factor IX, gene therapy
Subjects:QV Pharmacology, toxicology, pharmacy > QV 60-370 Pharmacology
QV Pharmacology, toxicology, pharmacy > QV 38 Pharmacogenetics
W Health professions > W 74-80 Medical economics. Health care costs
W Health professions > W 84 Health services. Quality of health care
W Health professions > W 100-275 Medical, dental and pharmaceutical service plans
WB Practice of medicine > WB 300-962 Therapeutics
WH Hemic and lymphatic systems
Language:English
Series Name:Decision Support Document for the Austrian Appraisal Board 002
Deposited on:30 Apr 2025 12:10
Last Modified:30 Apr 2025 12:10

Repository Staff Only: item control page