MONTREAL, February 25, 2025 – On February 20, Pfizer announced that it was stopping development of its hemophilia B gene therapy, Beqvez. It cited “soft demand from patients and their doctors.”
Beqvez was approved by Health Canada in December 2023 and recommended for reimbursement (with conditions) by Canada’s Drug Agency in March 2024. Negotiations had been underway with the pan-Canadian Pharmaceutical Alliance to set a price for public health plans. The initial list price was $4.7 million per single dose. Beqvez was being developed in partnership with Spark Therapeutics. There is no indication at this time that Spark will look for a new partner.
Meanwhile, Canadian price negotiations continue for CSL’s hemophilia gene B therapy, Hemgenix. Regeneron’s CRISPR-based gene insertion therapy for hemophilia B is starting phase 1 clinical trials in three Canadian centres: McMaster University in Hamilton, St. Michael’s Hospital in Toronto and the McGill University Hospital Centre in Montreal.
HEMOPHILIA A
In December 2024, Pfizer announced its was withdrawing from its partnership with Sangamo to develop the AAV-based gene therapy, giroctogeneb fitelparvovec, for hemophilia A. This means a stop to the phase 3 trial of 63 adults, 18 to 64 years. Also in December, Roche announced it was discontinuing further development of Spark’s experimental hemophilia A gene therapy, dirloctocogene samoparvovec, also known as SPK-8011.
In August 2024 Biomarin announced it would limit marketing of its hemophilia A gene therapy, Roctavian, to the U.S., Germany and Italy. No regulatory submission will be made to Health Canada.
These three gene therapies are based on an adeno associated viral vector to deliver the factor VIII gene therapy to the liver. A key drawback to this technology has turned out to be declining FVIII expression year-over-year.
Hope for successful hemophilia A gene therapies will now turn to next-generation approaches; however, these are several years away.
For more information, see the CHS Gene Therapy Education Program at www.hemophilia.ca/gene-therapy.
