The move comes weeks after Pfizer terminated its partnership with Sangamo Therapeutics for another hemophilia gene therapy.
Pfizer will no longer develop and commercialize its hemophilia B gene therapy Beqvez across all of its global markets, according to multiple media reports Thursday.
In a statement to Nikkei Asia, the pharma said that the decision was due to “the limited interest patients and their doctors have demonstrated in hemophilia gene therapies.” Beqvez, a one-time treatment, was approved by the FDA in April 2024 and was given a list price of $3.5 million per dose at launch.
Beqvez’s discontinuation could be a sign that Pfizer is distancing itself from hemophilia gene therapies. At the end of 2024, the pharma walked away from its hemophilia A co-development pact with Sangamo Therapeutics forgiroctocogene fitelparvovec, designed to restore patients’ ability to produce Factor VIII, a core clotting protein.
Pfizer did not explain at the time why it was abandoning Sangamo and its gene therapy, only noting that the move reflected the pharma’s decision not to proceed with regulatory and commercial activities for giroctocogene fitelparvovec, Sangamo disclosed at the time.
Outside of gene therapies, Pfizer seems to still see promise in hemophilia as a disease target. According to reporting from Reuters on Thursday, the pharma will continue to put money and time into Hympavzi, an IgG1 monoclonal antibody that helps promote clotting, which the FDA in October 2024 cleared for hemophilia A and B in adults and adolescents. The drug is designed to be administered subcutaneously once per week.
Pfizer’s move away from hemophilia gene therapies could be a sign of broader problems with the space. Roctavian, owned by BioMarin, became the industry’s first-ever gene therapy approved for hemophilia A in June 2023—only for the $2.9-million medicine to run into a largely unreceptive market. Following an underwhelming launch, the biotech in August 2024 rightsized its commercial efforts for Roctavian, focusing just on the U.S., German and Italian markets, where the gene therapy is approved and reimbursed.
In its most recent business report, covering the fourth quarter and full-year 2024, BioMarin reported $26 million in sales for Roctavian last year.
Elsewhere in the hemophilia space, Novo Nordisk earlier this month reported that its bispecific antibody Mim8 reduced bleeding events in pediatric patients. The pharma is preparing for regulatory submissions for Mim8 this year, which it will back with additional Phase III data underlining the antibody’s efficacy in adults and adolescents. Like Hympavzi, Mim8 is not a one-time treatment; instead, it can be administered once per week, once every two weeks or once per month.
