Pfizer’s one-time haemophilia B gene therapy Durveqtix granted EC approval

26 Jul 2024
pmlive.com
Pfizer’s one-time haemophilia B gene therapy Durveqtix granted EC approval
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Source: PMLiVE
Pfizer’s haemophilia B gene therapy has been granted conditional marketing authorisation by the European Commission (EC) to treat adults with severe and moderately severe cases of the rare bleeding disorder.
Durveqtix (fidanacogene elaparvovec) has been specifically authorised for use in patients without a history of both factor IX (FIX) inhibitors and detectable antibodies to variant adeno-associated virus serotype Rh74.
Affecting more than 42,000 people globally, haemophilia B is a genetic condition resulting from a deficiency in blood-clotting FIX.
The standard of care for this patient population requires regular intravenous infusions of FIX replacement products to prevent or treat bleeding. However, many patients with moderate-to-severe cases remain at risk of spontaneous bleeding episodes, underscoring the need for new treatment options that provide sustained bleed protection.
Pfizer’s Durveqtix, which is already approved in the US as Beqvez, is also administered as an intravenous infusion but is designed to enable patients to produce FIX themselves after a one-time dose.
The EC’s decision on the therapy follows a recent recommendation from the European Medicines Agency’s human medicines committee and is supported by positive results from the late-stage BENEGENE-2 of men aged 18 to 62 years with moderately severe-to-severe haemophilia B.
The trial met its primary efficacy endpoint of non-inferiority and demonstrated a statistically significant decrease in annualised bleeding rate for total bleeds post-Durveqtix infusion compared to a routine FIX prophylaxis regimen, administered as part of usual care.
Efficacy also remained stable during year two to year four after treatment and Durveqtix was shown to be generally well-tolerated, with a safety profile consistent with phase 1/2 results.
Alexandre de Germay, chief international commercial officer and executive vice president at Pfizer, said: “There is a substantial medical and treatment burden for people with haemophilia 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.
“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 haemophilia B. These outcomes and their impact could become potentially transformative for haemophilia B care in the EU.”
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