Less than a year after approval, Pfizer is planning to discontinue its haemophilia B gene therapy fidanacogene elaparvovec (Beqvez) across all global markets, citing weak demand.
The treatment was launched with a price tag of $3.5m per dose, reports Medpage Today.
“Pfizer has made the decision to cease further development and commercialisation activities for several reasons, including the limited interest patients and their doctors have demonstrated in haemophilia gene therapies to date,” the company said.
The company added that it would focus its efforts on treatments like marstacimab (Hympavzi) – a non-factor treatment option – “that we believe will have the greatest impact on patients”.
That drug, an IgG1 monoclonal antibody that helps promote clotting, was approved by the FDA in October for haemophilia A and B in adults and adolescents.
The move comes after Pfizer terminated a global collaboration and licence agreement with Sangamo Therapeutics to develop giroctocogene fitelparvovec, an investigational gene therapy for the treatment of adults with moderately severe to severe haemophilia A.
Fidanacogene elaparvovec was approved by the FDA last year for adult men with moderate to severe haemophilia B currently treated with prophylactic factor IX, who have a history of life-threatening haemorrhage or repeated serious spontaneous bleeding episodes, and who do not have neutralising antibodies to the adeno-associated virus serotype Rh74var capsid.
Support for the approval came from the phase 3, single-arm, open-label BENEGENE-2 trial involving 45 men with moderate or severe haemophilia B (factor IX circulating activity ≤2%).
From week 12 to data cut-off after 1.8 years of follow-up, the annualised bleeding rate decreased from 4.5 during the lead-in period to 2.5 after a single administration of the gene therapy. Bleeds were eliminated in 60% of patients with fidanacogene elaparvovec, compared with 29% with prophylactic factor IX.
The withdrawal of fidanacogene elaparvovec leaves one gene therapy – etranacogene dezaparvovec (Hemgenix) – approved for treatment of haemophilia B. It was approved in 2022 for adults who currently use factor IX prophylaxis therapy for blood clotting, those who have or have had life-threatening haemorrhage, and those who have repeated serious spontaneous bleeding episodes.
Medpage Today article – Pfizer to Discontinue Its Haemophilia B Gene Therapy (Open access)
See more from MedicalBrief archives:
FDA green lights Pfizer’s drug for haemophilia A, B
Experimental haemophilia drug slows bleeding – Pfizer
World’s most expensive drug approved by the FDA at R60m a dose