Pfizer’s Gene Therapy for Rare Bleeding Disorder Gets FDA Approval

Pfizer’s gene therapy for hemophilia B received approval from the U.S. Food and Drug Administration on Friday, April 26, 2024. It is the second such therapy to be approved for the rare bleeding disorder which requires regular infusion of a blood-clotting protein.

Hemophilia is a condition where people suffer severe bleeding from injuries or surgeries because their blood lacks the proteins called clotting factors. The condition results from a fault in the gene that regulates the production of the proteins. Males are predominantly affected by the condition.

Pfizer’s therapy is a one-time medication branded as Beqvez. It is designed to stimulate the production of factor IX (FIX) protein by the patient’s body instead of the regular intravenous infusion multiple times a week or month to introduce FIX into the body.

Pfizer’s Beqvez will be available by prescription to eligible patients this quarter said the company’s spokesperson. The drugmaker has set the price of the drug at $3.5 million in the United States. This matches the price tag of rival gene therapy Hemgenix, produced by Australian drugmaker CSL Ltd.

High price tags continue to hold back gene therapy

Logistic issues, the high cost of gene therapies, and the prospect of potential treatment advances have held back the adoption of the first gene therapies for hemophilia. Also, the durability of single-dose gene therapies has remained an open debate. 

“[The demand for gene therapies is restricted by] how satisfied they (patients) are with their current treatment…and whether they want to dive right in, which not many do,” said the director of the Hemophilia and Thrombosis Center at the University of North Carolina, Nigel Key.

After a year, the single dose of Pfizer’s gene therapy was shown to remain as effective as standard-of-care protein infusions. Bleeding was completely eliminated in 60% of patients which is far higher than the 29% recorded in patients who received infusions. Pfizer is committed to monitoring the long-term durability and safety of the treatment over the next 15 years. 

According to government data, it is estimated that hemophilia B affects around 4 in every 100,000 males in the United States while hemophilia A, a related disorder, affects around 12 in every 100,000 males in the United States.