The US Food and Drug Administration recently greenlit Pfizer’s groundbreaking hemophilia B gene therapy, Beqvez (fidanacogene elaparvovec-dzkt), setting a precedent with its wholesale acquisition cost surpassing $3 million. Hemophilia B, a rare genetic bleeding disorder stemming from factor IX (FIX) protein deficiency, finds a potential remedy in Beqvez, offering a one-time treatment to adults with moderate to severe forms of the condition. Unlike conventional prophylactic intravenous infusions, Beqvez provides a functional FIX gene copy, enabling patients to autonomously produce the vital protein.
Prior to administration, patients must undergo screening for neutralizing antibodies to the adeno-associated virus (AAV) serotype Rh74var capsid, employed in delivering the therapy. Pfizer’s Chief US Commercial Officer, Aamir Malik, underscored Beqvez’s potential to revolutionize hemophilia care, emphasizing its prolonged bleed protection and value proposition due to its single-dose administration.
The FDA’s approval was anchored in the BENEGENE-2 Phase III trial, encompassing 45 adult male participants afflicted with moderately severe to severe hemophilia B. Notably, Beqvez demonstrated non-inferiority in annualized bleeding rates compared to standard FIX infusions over a six-month lead-in period. Adverse reactions were predominantly manageable, with elevated transaminases representing the most common side effect, and no fatalities were recorded. Pfizer commits to monitoring participants for up to 15 years to assess long-term safety and efficacy.
The $3.5 million price tag for Beqvez reflects Pfizer’s holistic valuation encompassing patient benefits, healthcare system savings, and societal impact, juxtaposed against the substantial annual expenses of conventional hemophilia B management, estimated at $1.1 million per patient. This pricing places Beqvez alongside CSL Behring’s Hemgenix and Orchard Therapeutics’ Lenmeldy, positioning it within the realm of the world’s most expensive drugs.
To further facilitate access, Pfizer introduces a warranty program offering refunds contingent on treatment durability, alongside copay assistance and comprehensive patient support services. Notably, Beqvez has already secured regulatory nods in Canada and awaits European Medicines Agency review.
Amidst the buzz surrounding Beqvez, Pfizer’s commitment to ensuring accessibility underscores its dedication to transforming hemophilia management. As gene therapies redefine treatment paradigms, Beqvez stands at the vanguard, heralding a new era of precision medicine for rare diseases.
