Pfizer's first gene therapy approved in the United States! Treating type B hemophilia

On Friday, April 26th, the Food and Drug Administration (FDA) of the United States announced the official approval of a new drug developed by Pfizer called Beqvez for the treatment of Hemophilia B. This is Pfizer's first gene therapy drug approved in the U.S. market. Hemophilia B is a rare genetic bleeding disorder that mainly affects males. It is caused by a lack or complete absence of a blood clotting protein called factor IX in the patient's body. The deficiency of this protein makes blood clotting difficult, leading to easy bruising and prolonged bleeding time in patients. In severe cases, patients may even experience spontaneous bleeding without obvious trauma. Pfizer's new drug Beqvez, as a one-time gene therapy, aims to enable patients to produce factor IX on their own, effectively preventing and controlling bleeding. Beqvez showed better results in late-stage trials compared to traditional treatments. Traditional treatment requires multiple weekly or monthly intravenous injections of factor IX. Dr. Adam Cuker, Director of the Hemophilia and Thrombosis Program at the University of Pennsylvania Medical Center, stated, "Many Hemophilia B patients feel disrupted in their lives due to the need for regular injections of factor IX, and they also experience spontaneous bleeding events, which can lead to joint pain and mobility issues. Beqvez, as a one-time treatment, is expected to reduce the burden on patients and treatment." According to the Hemophilia B Alliance, there are approximately over 7,000 Hemophilia B patients in the United States, but the actual number of affected individuals may be higher. As reported by the media, a Pfizer spokesperson revealed that the price of this drug is as high as $3.5 million before insurance and other discounts, making it one of the most expensive drugs in the U.S. market. However, the company has provided a quality assurance plan to ensure economic protection for patients receiving Beqvez treatment in case the desired effects are not achieved. Furthermore, Beqvez will compete with another drug called Hemgenix for treating Hemophilia B, which is produced by the Australian company CSL Behring and also received FDA approval in 2022. Hemgenix is priced at $3.5 million in the U.S. as well (before insurance and discounts). It is worth noting that some health experts have pointed out that the high cost of treatment and logistical issues limit the widespread adoption of such new therapies. Following the announcement, as of the time of writing, Pfizer's stock price has risen by 0.87%.