Pfizer has received U.S. FDA approval for Beqvez (fidanacogene elaparvovec-dzkt), a novel one-time gene therapy for adults suffering from moderate to severe hemophilia B.
The new treatment is approved for patients who are on routine factor IX (FIX) prophylaxis, those with a history of severe hemorrhage, or those experiencing repeated serious spontaneous bleeds, provided they do not have neutralizing antibodies against the AAVRh74var capsid, based on FDA-approved testing.
Beqvez is engineered to reduce the dependence on frequent FIX infusions, which are typically required multiple times per week or month under the current standard of care. Clinical studies have demonstrated that a single administration of Beqvez can effectively reduce bleeding incidents, with a recorded median annualized bleeding rate of zero in many cases, following up to three years of patient monitoring.
Hemophilia B, a rare genetic disorder, leads to inadequate blood clotting due to a deficiency in clotting factor IX, resulting in more frequent and severe bleeding episodes.
Pfizer has priced Beqvez at $3.5 million per treatment in the U.S. market, and is launching a warranty program to support the therapy’s rollout, aimed at maximizing accessibility and providing financial safeguards against potential efficacy failures. Recently, Lenmeldy, which won FDA approval back in March, knocked CSL's $3.5 million hemophilia B gene therapy to second place on the list of the world's most expensive drugs.
Beqvez was recently approved in Canada and is under review by the European Medicines Agency. Pfizer is also exploring gene therapy applications for other conditions, including hemophilia A and Duchenne muscular dystrophy.
