EC Approves Pfizer's One-Time Haemophilia B Gene Therapy Durveqtix

Pfizer has received conditional marketing authorization from the European Commission (EC) for its haemophilia B gene therapy, Durveqtix (fidanacogene elaparvovec). This approval is for adults with severe and moderately severe cases of the rare bleeding disorder. The therapy is designated for patients without a history of factor IX (FIX) inhibitors and without detectable antibodies to the Rh74 variant adeno-associated virus serotype.

Haemophilia B is a genetic disorder affecting over 42,000 individuals worldwide. It is characterized by a deficiency in the blood-clotting protein FIX. The current standard of care involves regular intravenous infusions of FIX replacement products to manage and prevent bleeding episodes. Despite this regimen, many patients with moderate-to-severe haemophilia B still face the risk of spontaneous bleeding, highlighting an urgent need for more effective treatment options.

Durveqtix, which is also approved in the United States under the name Beqvez, offers a new approach. Administered via a one-time intravenous infusion, it is designed to enable patients to produce FIX internally. This internal production aims to provide sustained protection against bleeding episodes.

The EC's decision follows a recommendation from the European Medicines Agency’s human medicines committee and is based on positive outcomes from the late-stage BENEGENE-2 trial. This study included men aged 18 to 62 years with moderately severe-to-severe haemophilia B. The trial met its primary efficacy endpoint of non-inferiority and showed a significant reduction in the annualized bleeding rate post-Durveqtix infusion compared to routine FIX prophylaxis.

Further, the efficacy of Durveqtix remained stable from the second year to the fourth year post-treatment. The therapy was generally well-tolerated, with a safety profile consistent with earlier phase 1/2 trial results.

Alexandre de Germay, Pfizer’s Chief International Commercial Officer and Executive Vice President, commented on the significance of this approval. He emphasized the substantial burden faced by haemophilia B patients who rely on current standard care, which involves frequent infusions and the persistent risk of breakthrough bleeds that can cause pain and restricted mobility. According to de Germay, Durveqtix has the potential to offer long-term bleed protection with a single dose, reducing or even eliminating bleeding episodes for suitable patients. He underscored the potential transformative impact this could have on haemophilia B care in the European Union.

The approval of Durveqtix marks a significant advancement in the treatment of haemophilia B and offers hope for improved quality of life for those affected by this condition.