Janice R. Andrews
Pfizer has reached a critical milestone by obtaining FDA approval for its gene treatment, Beqvez (fidanacogene elaparvovec-dzkt), which is intended for people with hemophilia B.
Pfizer’s move into the gene therapy space comes a decade after its original partnership with Spark Therapeutics. Beqvez is the second FDA-approved gene treatment for hemophilia B, following Hemgenix by CSL and uniQure, which launched in 2022 at a cost of $3.5 million.
Despite the potential to provide a lower price, Pfizer has decided to match its competitor’s cost, with Beqvez priced at $3.5 million per dose. A spokesman from Pfizer confirmed to Fierce Pharma that the therapy will be available this quarter.
With an estimated 33,000 boys affected by hemophilia in the United States, Pfizer’s Beqvez shows potential for improving treatment outcomes. Hemophilia B, while less common than Hemophilia A, is a substantial healthcare challenge since it affects people who lack the factor IX protein, which is necessary for blood coagulation.
Pfizer’s Beqvez not only meets the medical demands of hemophilia B patients, but it also promises to reduce the financial burden associated with current standard-of-care therapies. The therapy represents a break from frequent intravenous transfusions, which can cost patients up to $1.1 million per year. Pfizer promotes Beqvez’s long-term value proposition, citing possible cost savings to the healthcare system.
Furthermore, Pfizer provides a warranty program to provide financial protection against efficacy failures. This initiative demonstrates Pfizer’s commitment to assuring the efficacy of its gene therapy.
Pfizer’s venture into gene therapy goes beyond hemophilia B, with phase 3 trials underway for hemophilia A and Duchenne muscular dystrophy. Additionally, the company is working on an anti-tissue factor pathway inhibitor for hemophilia A and B.
As Pfizer continues to innovate in gene therapy, it recently sold a portfolio of preclinical rare illness gene medicines to AstraZeneca, highlighting its strategic focus on this therapeutic area.
The FDA’s clearance of Beqvez demonstrates the transformative potential of gene therapy in treating rare genetic illnesses, providing hope to both patients and caretakers.
