EC Grants Conditional Approval for Pfizer's Hemophilia B Gene Therapy

The European Commission (EC) has granted conditional marketing authorisation for Pfizer’s DURVEQTIX (fidanacogene elaparvovec), a one-time gene therapy designed for adults with severe and moderately severe haemophilia B. This therapy is intended for patients who do not have a history of factor IX inhibitors and lack detectable antibodies to the variant AAV serotype Rh74.

DURVEQTIX enables patients with haemophilia B to produce factor IX internally, thereby eliminating the necessity for frequent intravenous infusions that are typically required weekly or biweekly. The approval for DURVEQTIX is underpinned by data from the Phase III BENEGENE-2 clinical trial, which evaluated the therapy's effectiveness and safety in adult male participants aged between 18 and 62 years who have haemophilia B. The primary efficacy endpoint of the study was successfully met, demonstrating a statistically significant reduction in the annualised bleeding rate for total bleeds following treatment with DURVEQTIX compared to the standard prophylaxis regimen.

The clinical trial results indicated that the efficacy of DURVEQTIX remained consistent from the second to the fourth year after administration. Moreover, the therapy was generally well-tolerated by participants, with a safety profile in line with the observations from Phase I/II data. This conditional marketing authorisation applies across the 27 EU member states as well as Iceland, Liechtenstein, and Norway. Additionally, the therapy has also received approval from the US Food and Drug Administration (FDA) and Health Canada, where it is marketed under the name BEQVEZ.

Pfizer has also achieved positive data from a Phase III programme for gene therapy in haemophilia A, specifically for giroctocogene fitelparvovec. Furthermore, a Phase III trial is currently in progress for marstacimab, an investigational treatment aimed at individuals with haemophilia A and B, both with and without inhibitors.

Alexandre de Germay, Pfizer’s chief international commercial officer and executive vice-president, remarked on the significant medical and treatment challenges faced by people with haemophilia B who receive the current standard of care. This includes frequent infusions and the persistent risk of breakthrough bleeds, which can result in pain and limited mobility. He highlighted that DURVEQTIX offers a potential long-term solution by providing bleed protection with a single dose, thereby reducing or possibly eliminating bleeding episodes for suitable patients. De Germay believes that these promising outcomes could bring transformative changes to haemophilia B care within the European Union.

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