Financial Services, April 27 (Editor: Niu Zhanlin) On Friday EST, US pharmaceutical giant Pfizer announced that the US Food and Drug Administration (FDA) has approved the company's gene therapy for hemophilia, making it the company's first gene therapy approved in the US. In the US stock market, Pfizer's stock price rose nearly 1%.

Hemophilia is a congenital hemorrhagic disease. It is generally caused by abnormal blood clotting factors, and once it develops, it lasts a lifetime. It is characterized by prolonged blood clotting time and a lifelong tendency to bleed after minor trauma. “Spontaneous” bleeding can occur even in critically ill patients without obvious trauma.

This gene therapy approved by the FDA is called Beqvez and is used in adults with moderate to severe hemophilia B. Beqvez is a one-off treatment that enables patients to produce clotting factor IX and prevent bleeding. Without this protein known as coagulation factor IX, hemophilia B patients are more likely to be injured, bleed more frequently, and last longer.

In trials, it was shown to be superior to standard treatments, reducing the need for regular weekly or monthly intravenous medication. Beqvez has the potential to change the lives of patients with hemophilia B by reducing the burden of medical intervention and treatment.

A Pfizer spokesperson said the treatment will be offered by prescription to eligible patients this quarter. Excluding insurance and other discounts, the price of each dose is as high as 3.5 million US dollars, making it one of the most expensive drugs in the US so far.

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

Cuker added that Pfizer's medicines have the potential to have a transformative impact on patients by reducing long-term care and treatment burdens.

According to the World Federation of Hemophilia, more than 38,000 people around the world have hemophilia B, yet few people can afford Beqvez.

This approval is an important step for Pfizer, which is trying to gain a foothold after the rapid decline in the COVID-19 business last year. The company is already betting heavily on cancer drugs and treatments in the field of other diseases to help reverse its decline in business.

Pfizer is one of the few companies investing in the rapidly growing field of gene and cell therapy. These one-off, high-cost treatments target patients' genetic origins or cells to cure or significantly alter the course of the disease. Some health experts expect these treatments to replace traditional lifelong treatments people use to manage chronic diseases.

Beqvez will compete with Australia's CSL Behring's Hemgenix, the first FDA-approved haemophilia B gene therapy, launched in 2022 at a price of $3.5 million per dose.

Notably, some health experts say factors such as high costs and logistics issues have limited the adoption of Hemgenix and another approved haemophilia A gene therapy.