Montreal, August 10, 2023 – Gene therapies for hemophilia A and B continue to move closer to becoming reality.
Health Canada is currently evaluating two gene therapies for hemophilia B.
The first is etranacogene dezaparvovec, manufactured by CSL Behring and marketed in the U.S. and Europe as Hemgenix. A decision on approval in Canada could come as early as the fourth quarter of 2023. Hemgenix was approved by the U.S. Food and Drug Administarion (FDA) in November 2022 and by the European Medicines Agency (EMA) in February 2023.
Results from the Phase 3 HOPE-B study were published in the New England Journal of Medicine in February 2023 (1).
The second is fidanacogene elaparvovec, manufactured by Pfizer. This gene therapy is under review by Health Canada, the FDA and the EMA. A decision in Canada could come late in 2023 or early in 2024.
Safety and efficacy data from the Phase 3 BENEGENE-2 trial were presented at the recent Congress of the International Society of Hemostasis and Thrombosis (ISTH), held in Montreal in June.
Both these gene therapies will be undergoing health technology assessments in the coming months by INESSS for Quebec and by CADTH for the rest of Canada. The Canadian Hemophilia Society is making submissions to present the patient perspective. Parts of the submissions will present the ideas expressed by the community in the summer 2022 survey on attitudes to gene therapy, and the current survey of the lived experience of those with hemophilia B. To participate in the survey, please click on the following link: www.surveymonkey.com/r/gene-therapy-FIX.
Meanwhile, Biomarin’s Roctavian, a gene therapy for hemophilia A, was licensed by the U.S. FDA in June 2023, after an earlier approval in August 2022 by the EMA. Biomarin has yet to file for approval in Canada. No one has been treated post-marketing in Europe; at least one person has been treated in the U.S.
The latest data on Roctavian, presented in June at the ISTH Congress, continue to show slowly decreasing FVIII expression from year to year and wide variability in response from person to person.
(1) Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B, Steven W. Pipe et al, N Engl J Med 2023; 388:706-718 DOI: 10.1056/NEJMoa2211644