EU clears Pfizer’s haemophilia B gene therapy Durveqtix
26 Jul 2024
The European Commission granted conditional marketing authorisation to Pfizer’s Durveqtix (fidanacogene neparvovec) to treat adults with moderate-to-severe haemophilia B, making it the second gene therapy approved for the condition in the EU after CSL and uniQure's Hemogenix (etranacogene dezaparvovec). Durveqtix is designed to deliver a functional copy of a high-activity factor IX gene variant, enabling patients to endogenously produce the coagulation factor.
Hot on the heels of approvals in Canada and the US under the name Beqvez, the European Commission’s clearance of the gene therapy follows a positive recommendation by the European drug advisory body in May. Highlighting the unmet treatment burden for patients currently receiving standard of care, Alexandre de Germay, chief international commercial officer at Pfizer, said Durveqtix’s “potential to offer long-term bleed protection in a one-time dose” could be “transformative for haemophilia B care.”
The EU approval was based on the Phase III BENEGENE-2 study, where the gene therapy met its primary efficacy endpoint of non-inferiority, showing a statistically significant reduction in annualised bleeding rate (ABR) for total bleeds compared with standard prophylaxis using factor IX. Furthermore, the efficacy was sustained from year two to year four post-treatment.
Pfizer has set Beqvez’s price at $3.5 million for the one-time treatment in the US, to align with the similarly-priced Hemgenix. The drugmaker, however, is offering a “warranty” programme on its therapy to insure US payers against the risk of efficacy failure. Meanwhile, Pfizer, in partnership with Sangamo Therapeutics, is also investigating the gene therapy giroctocogene tiparvovec (giro-vec), for haemophilia A, with positive results in the Phase III AFFINE study.
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