FDA Approves Pfizer’s One-Time Gene Therapy for Hemophilia B: $3.5 Million per Dose

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FDA Approves Pfizer’s One-Time Gene Therapy for Hemophilia B: Priced at $3.5 Million per Dose

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FDA Approves Pfizer’s One-Time Gene Therapy for Hemophilia B: Priced at $3.5 Million per Dose

On April 26, Pfizer, the American pharmaceutical giant, announced that the U.S. Food and Drug Administration (FDA) had approved the company’s gene therapy for treating hemophilia, making it the company’s first gene therapy to receive approval in the United States. Pfizer’s stock rose nearly 1% during trading.

Hemophilia is a congenital bleeding disorder typically caused by abnormalities in clotting factors, leading to a lifelong condition where patients have a tendency to bleed even with minor injuries or spontaneously without apparent trauma.

The FDA-approved gene therapy, named Beqvez, is intended for use in adult patients with moderate to severe hemophilia B. Beqvez is a one-time treatment that enables patients to produce clotting factor IX and prevent bleeding. Without this protein, known as factor IX, patients with hemophilia B are more prone to injury, with more frequent and prolonged bleeding episodes.

In trials, Beqvez has been shown to be superior to standard treatments, reducing the need for weekly or monthly intravenous injections of medication. By reducing the need for medical interventions and treatment burdens, Beqvez has the potential to change the lives of hemophilia B patients.

A spokesperson for Pfizer stated that the therapy will be available to eligible patients through prescriptions in the current quarter. The price per dose is as high as $3.5 million without insurance or other discounts, making it one of the most expensive drugs in the United States to date.

Dr. Adam Cuker, Director of the Hemophilia and Thrombosis Program at the University of Pennsylvania Medical School, said on Friday, “Many hemophilia B patients face the costs of regular infusions of factor IX and disruptions to their lifestyle, as well as spontaneous bleeding events, which can lead to painful joint damage and mobility issues.”

Cuker added that Pfizer’s drug has the potential to bring about transformative impacts by reducing long-term medical and treatment burdens for patients.

According to data from the World Federation of Hemophilia, there are over 38,000 people worldwide with hemophilia B, but very few may be able to afford Beqvez.

This approval marks an important step for Pfizer as the company seeks to regain its footing after a sharp decline in its business due to the COVID-19 pandemic last year. The company has been heavily investing in treatments for cancer and other diseases, aiming to reverse its business decline.

Pfizer is one of the few companies investing in the rapidly growing field of gene and cell therapies, which are one-time, high-cost treatments targeting patients’ genetic origins or cells to cure or significantly alter the course of a disease. Some health experts predict that these therapies will replace traditional lifelong treatments used to manage chronic diseases.

Beqvez will compete with Hemgenix from Australia’s CSL Behring, which was the first FDA-approved gene therapy for hemophilia B, launched in 2022 at a price of $3.5 million per dose.

It is worth noting that some health experts have expressed concerns that factors such as high costs and logistical issues have limited the adoption of Hemgenix and another approved gene therapy for hemophilia A.

FDA Approves Pfizer’s One-Time Gene Therapy for Hemophilia B: Priced at $3.5 Million per Dose

(source:internet, reference only)

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