On Friday, April 26, the US Food and Drug Administration (FDA) announced that it has officially approved a new drug called Beqvez developed by Pfizer to treat hemophilia B. According to reports, this is the first gene therapy drug approved by Pfizer in the US market.
Hemophilia B is a rare hereditary bleeding disorder that mainly affects men. The cause is a lack or complete absence of a blood clotting protein called factor IX in the patient's body. This lack of protein makes it difficult for blood to clot, making patients prone to bruising and prolonged bleeding time. In severe cases, patients may even experience spontaneous bleeding without significant trauma.
Pfizer's new drug, Beqvez, is a single-use gene therapy designed to enable patients to produce factor IX on their own, thereby effectively preventing and controlling bleeding. Beqvez showed better results than traditional treatments in advanced trials. Traditional treatment requires intravenous administration of factor IX every week or several times a month.
Dr. Adam Cuker, director of the Comprehensive Medicine and Hemophilia and Thrombosis Program at the University of Pennsylvania, said, “Many people with hemophilia B feel that their lives are disrupted because they need regular injections of factor IX, and they also experience spontaneous bleeding events, which can cause joint pain and mobility problems. As a one-time treatment, Beqvez is expected to reduce the burden on patients and treatment.”
According to the Hemophilia B Alliance, there are about 7,000 people with type B hemophilia in the US, but the actual number of people with the disease may be higher.
According to media reports, a Pfizer spokesperson revealed that the price of this drug was as high as 3.5 million US dollars before insurance and other discounts were deducted, making it one of the most expensive drugs on the US market. However, the company provides a warranty plan to protect patients treated with Beqvez and ensure that they receive financial protection when results are not up to standard.
Additionally, Beqvez will compete with Hemgenix, another drug to treat type B hemophilia, which is produced by the Australian company CSL Behring and was also approved by the FDA in 2022. Hemgenix's price in the US is also $3.5 million (before insurance and discounts). Notably, according to some health experts, high treatment costs and logistical issues have limited the spread of these new treatments.
After the news was announced, as of press time, Pfizer's stock price had risen 0.87%.
