Pfizer’s DURVEQTIX approved as a one-time gene therapy for adults with Hemophilia B

Pfizer recently announced that the European Commission had granted conditional marketing authorisation for DURVEQTIX which is a gene therapy for the treatment of severe and moderately severe haemophilia B in adult patients without a history of factor IX inhibitors and without detectable antibodies to variant AAV serotype Rh74.

“There is a substantial medical and treatment burden for people with hemophilia B that receive standard of care today, with frequent infusions and many remaining at risk of breakthrough bleeds that can lead to pain and restricted mobility,” said Alexandre de Germay, Chief International Commercial Officer and Executive Vice President, Pfizer. “DURVEQTIX has shown the potential to offer long-term bleed protection in a one-time dose, reducing or eliminating bleeds for the appropriate patients with hemophilia B.

Hemophilia B is a rare genetic bleeding disorder that prevents normal blood clotting because of a deficiency in FIX that causes those with the disease to bleed more frequently and longer than others. The standard of care for hemophilia B treatment is prophylactic infusions of FIX replacement therapy that temporarily replace or supplement low levels of blood-clotting factor. Despite prophylaxis and regular intravenous infusions, many people living with moderate to severe hemophilia B are at risk of spontaneous bleeding episodes.

The conditional marketing authorization is based on results from the pivotal Phase 3 BENEGENE-2 study evaluating the efficacy and safety of DURVEQTIX in adult male participants with moderately severe to severe hemophilia B. BENEGENE-2 met its primary efficacy endpoint of non-inferiority and demonstrated a statistically significant decrease in annualized bleeding rate for total bleed post-DURVEQTIX infusion versus prophylaxis regimen with FIX, administered as part of usual care. Efficacy, based on ABR, also remained stable during year two to year four after treatment. DURVEQTIX was generally well-tolerated, with a safety profile consistent with Phase 1/2 results.

 

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Related Topics and Keywords

(fidanacogene elaparvovec, DURVEQTIX®, Hemophilia B