Pfizer's bet on gene therapies in haemophilia has been a bust

Yahoo

27-02-2025

Pfizer has ended the global commercialisation of its haemophilia B (factor IX deficiency) gene therapy Beqvez, citing soft demand for gene therapies among haemophilia patients and physicians. This announcement comes after other news that Pfizer ended its partnership with Sangamo Therapeutics for the haemophilia A (factor XIII deficiency) gene therapy giroctocogen fitelparvovec in December 2024. The therapy had received positive Phase III results and was likely to receive US Food and Drug Administration (FDA) approval. However, Pfizer determined that the cost of launching and commercialising the drug would exceed its anticipated sales. Both Biomarin, which markets the haemophilia A gene therapy Roctavian, and CSL Behring, which markets the haemophilia B gene therapy Hemgenix, have announced that the uptake and sales for their respective therapies have been below expectations.
The commercial failure of these adeno-associated virus (AAV) gene therapy programmes has come as a surprise to those who were familiar with the opinions of patients and key opinion leaders during clinical trials, as they were generally very positive. However, opinions changed when longer-term efficacy and safety data was published, as many patients who received haemophilia A gene therapies eventually returned to prophylactic treatment as expression of factor XIII reduced over time. Even if patients are interested in these therapies, they may be ineligible. Patients who receive Roctavian must test negative for the AAV serotype used in the gene therapy. Patients with factor inhibitors or certain liver conditions were also excluded from haemophilia gene therapy trials. Even if the patient is eligible, the high list price has discouraged payers from reimbursing the therapies, limiting uptake. Patients may decide to wait for a potential future generation of gene therapies with better safety and efficacy, especially considering the fact that the current prophylaxis treatments have a lower disease burden than previous options. Roche's Hemlibra can be dosed subcutaneously once per month, which is a significant improvement over the multiple infusions per week that were common when these gene therapy programmes started.
Pfizer has not abandoned the haemophilia market completely. Instead, it has shifted its focus to Hympavzi, an antibody that received FDA approval for the treatment of haemophilia A and B without factor inhibitors, which is dosed subcutaneously once per week. However, both Pfizer and Roche will closely be watching Sanofi's fitusiran, a small interfering ribonucleic acid therapy that is currently in preregistration for haemophilia A and B with or without inhibitors, and which can be dosed as infrequently as once every two months, significantly reducing the treatment burden on patients.
Pfizer's exit from the gene therapy space has reduced the competition for eligible interested patients. Leading data and analytics company GlobalData's analyst consensus forecast predicts that sales for Roctavian will reach $337m in 2030 while Hympavzi will reach sales of $251m in the same year. It is possible that innovative reimbursement strategies such as outcome-based agreements could speed up their slow start in sales. It remains to be seen whether or not Pfizer's strategy is the correct one.
"Pfizer's bet on gene therapies in haemophilia has been a bust" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand.
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