The Food and Drug Administration (FDA) announced its approval of Pfizer’s treatment for a rare genetic bleeding disorder, marking the company’s first gene therapy to gain clearance in the U.S.
The drug, to be marketed as Beqvez, received the green light from the agency for adults dealing with moderate to severe hemophilia B who meet specific criteria.
According to a Pfizer spokesperson speaking to CNBC, the treatment will be available by prescription to eligible patients this quarter. However, it comes with a substantial price tag of $3.5 million before insurance and other rebates, positioning it as one of the costliest drugs in the United States.
An advocacy group estimates that over 7,000 individuals in the U.S. are grappling with this debilitating condition, predominantly affecting men. Hemophilia B occurs due to insufficient levels of a protein crucial for blood clotting, leading to increased bruising and prolonged bleeding.
Beqvez represents a one-time treatment aiming to enable patients to produce the essential protein themselves, thereby managing and preventing bleeding episodes. Clinical trials demonstrated the drug’s superiority over the conventional treatment method, which often involves frequent protein infusions via veins.
Pfizer’s achievement is significant as the company seeks to rebound from the decline in its COVID-related business last year. It is now focusing on cancer drugs and therapies for other diseases to revitalize its operations.
The approval underscores Pfizer’s investment in gene and cell therapies, a field expected to revolutionize disease management by offering one-time, albeit costly, treatments that target genetic or cellular sources of illness.
Pfizer acquired the rights to develop and market Beqvez from Spark Therapeutics in 2014. Additionally, the company is introducing a warranty program to provide financial protection to patients receiving Beqvez.
Beqvez will enter the market alongside Hemgenix from Australia-based CSL Behring, another FDA-approved treatment for hemophilia B with a comparable price point. However, challenges such as high costs and logistical issues have hindered the widespread adoption of gene therapies for hemophilia thus far.
In addition to Beqvez, Pfizer is seeking FDA approval for marstacimab, an experimental antibody intended to treat both hemophilia A and B. The company is also engaged in the development of a gene therapy for Duchenne muscular dystrophy, a progressive genetic disorder affecting muscle strength.
