Three months after bagging FDA approval, Pfizer’s hemophilia B gene therapy has been cleared to expand its reach across the pond.
The therapy, which is branded as Beqvez in the U.S., received a conditional marketing authorization from the European Commission under the moniker Durveqtix. Specifically, European regulators approved the treatment’s use in adults with severe and moderately severe hemophilia B who don’t have history of treatment with factor IX inhibitors or detectable antibodies to variant AAV serotype Rh74.
Now, Pfizer’s offering is poised to help patients across the region produce the blood-clotting protein factor IX through a one-time dose, as opposed to the standard of care of multiple weekly or biweekly intravenous infusions.
“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,” Pfizer’s chief international commercial officer, Alexandre de Germay, said in a release. “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. These outcomes and their impact could become potentially transformative for hemophilia B care in the European Union.”
Pfizer first took on Durveqtix from Spark Therapeutics in 2014 for $20 million upfront and $260 million in potential milestones, marking the drugmaker’s foray into gene therapy. The latest approval, which follows a recommendation from the European Medicines Agency, marks the third for the therapy this year after Canada’s nod in January and the FDA’s in April.
But CSL and uniQure beat Pfizer to the punch with their Hemgenix, the world’s first gene therapy for the blood disease. Upon its 2022 FDA approval, that treatment became the most expensive drug in the world at the time with a $3.5 million price tag.
Pfizer charges the same amount for its rival in the U.S, a price point that represents a “compelling value proposition that can deliver savings for the healthcare system when you consider potential long-term benefits that would offset costs that are associated with chronic disease management today,” a spokesperson previously told Fierce Pharma.
Outside of Durveqtix, Pfizer is looking to expand its hemophilia portfolio with its giroctocogene fitelparvovec. That gene therapy proved superior to standard-of-care treatment for hemophilia A in a recently reported phase 3 study, achieving a “statistically significant” mean reduction in annualized bleed rate compared to patients’ baseline characteristics.
