EU Approves Pfizer's Durveqtix for Hemophilia B

Pfizer has received conditional marketing authorization from the European Commission for its gene therapy, Durveqtix (fidanacogene neparvovec), aimed at treating adults with moderate-to-severe haemophilia B. This approval marks the second gene therapy for haemophilia B in the European Union, following CSL and uniQure's Hemogenix (etranacogene dezaparvovec).

Durveqtix works by delivering a functional copy of a high-activity factor IX gene variant, which enables patients to produce the necessary coagulation factor internally. This gene therapy has already seen approvals in Canada and the United States under the name Beqvez, and its clearance in the EU follows a positive recommendation from the European drug advisory body given in May.

Pfizer's chief international commercial officer, Alexandre de Germay, emphasized the significant unmet needs of patients receiving the current standard of care for haemophilia B. He highlighted that Durveqtix could potentially offer long-term bleed protection with just a single dose, which could be transformative for the treatment of haemophilia B.

The European Commission's approval was based on results from the Phase III BENEGENE-2 study. In this study, Durveqtix achieved its primary efficacy endpoint of non-inferiority, demonstrating a statistically significant reduction in the annualized bleeding rate (ABR) for total bleeds compared to the standard prophylaxis using factor IX. Additionally, the efficacy of Durveqtix was maintained from the second year up to the fourth year post-treatment.

In the United States, Pfizer has priced Beqvez at $3.5 million for the one-time treatment, aligning it with the similarly priced Hemogenix. To address concerns about efficacy, Pfizer is offering a "warranty" program for US payers, which insures against the risk of the therapy failing to perform as expected.

Moreover, Pfizer is collaborating with Sangamo Therapeutics on another gene therapy, giroctocogene tiparvovec (giro-vec), for the treatment of haemophilia A. This therapy has shown positive results in the Phase III AFFINE study, indicating ongoing efforts to expand gene therapy options for different types of haemophilia.

In conclusion, the European Commission's approval of Durveqtix signifies a significant advancement in the treatment of haemophilia B, providing a novel option that could significantly improve the quality of life for patients. The therapy's entry into the market, at a price comparable to existing gene therapies, also highlights the broader trend toward innovative treatments in the realm of genetic disorders.