Pfizer recently announced the termination of the global development and commercialization of its hemophilia B gene therapy, Beqvez. This decision marks Pfizer’s further withdrawal from the gene therapy field.  

Reasons Behind This Decision:

1. Poor Market Performance:

   Since receiving FDA approval in April 2024, Beqvez has failed to meet market expectations. Its high price ($3.5 million per patient) has deterred both patients and insurance providers, making market adoption challenging.  

2. Intense Market Competition:

   The hemophilia B treatment market is already occupied by other gene therapies, such as CSL Behring’s Hemgenix and BioMarin’s Roctavian, which have taken up market share and further squeezed Beqvez’s commercial potential.  

Pfizer’s Prior Moves in the Gene Therapy Field:

-December 2024: Pfizer terminated its collaboration with Sangamo Therapeutics on the hemophilia A gene therapy giroctocogene fitelparvovec (giro-vec), despite positive Phase III clinical trial results and an imminent regulatory filing.  

- September 2024: Pfizer withdrew all batches of its sickle cell disease therapy Oxbryta from the market and discontinued all related clinical trials and expanded access programs. This decision was based on recent clinical data showing that the overall benefit of Oxbryta no longer outweighed its risks, particularly concerning the imbalance between vaso-occlusive crises and mortality rates.  

- June 2024: Pfizer’s Duchenne muscular dystrophy (DMD) gene therapy, fordadistrogene movaparvovec (PF-06939926), failed in its Phase III CIFFREO trial, as it did not meet the primary endpoint.  

- July 2023: Pfizer sold its early-stage gene therapy pipeline to AstraZeneca’s rare disease unit, Alexion, for up to $1 billion.  

Strategic Implications:

These actions highlight Pfizer’s strategic retreat from gene therapy. First, the high costs of development and pricing of gene therapies limit their market acceptance. Second, both patients and physicians remain cautious about adopting emerging gene therapies, affecting their commercial adoption.  

Pfizer’s withdrawal underscores the challenges in the commercialization of gene therapies, serving as a reminder for the industry to proceed cautiously when advancing these treatments into practical applications.