On Friday Eastern Time, US pharmaceutical giant Pfizer announced that the US Food and Drug Administration (FDA) has approved a gene therapy for treating hemophilia, making it the first gene therapy approved by the company in the United States. During the US stock market session, Pfizer's stock price rose nearly 1%.
Hemophilia is a congenital hemorrhagic disease, usually caused by abnormal coagulation factors, and once it occurs, it will accompany for a lifetime. Its characteristic is prolonged coagulation time, with a lifelong tendency for mild post-traumatic bleeding, and severe patients may experience "spontaneous" bleeding even without obvious trauma.
The FDA approved gene therapy is called Beqvez and is used in adults with moderate to severe hemophilia B. Beqvez is a one-time treatment method that allows patients to produce coagulation factor IX and prevent bleeding. Without this protein called coagulation factor IX, hemophilia B patients would be more prone to injury, bleeding more frequently, and lasting longer.
In the experiment, it was proven to be superior to standard treatment methods, reducing the need for regular intravenous injections of medication on a weekly or monthly basis. Beqvez has the potential to change the lives of hemophilia B patients by reducing the burden of medical intervention and treatment.
A spokesperson for Pfizer stated that the therapy will be provided to eligible patients through prescription this quarter, with a price of up to $3.5 million per dose, excluding insurance and other discounts, making it one of the most expensive drugs in the United States to date.
Adam Cuker, Director of the Hemophilia Thrombosis Integrated Program at the University of Pennsylvania School of Medicine, said on Friday, "Many hemophilia B patients face the cost of regular infusion of Factor IX and lifestyle disruptions, as well as spontaneous bleeding events, which can lead to painful joint injuries and mobility problems."
Cuker added that Pfizer's drugs have the potential to bring transformative effects to patients by reducing long-term medical and treatment burdens.
According to the World Hemophilia Federation, there are over 38000 people worldwide suffering from hemophilia B, but few can afford Beqvez.
This approval is an important step for Pfizer, which is trying to regain its footing after the rapid decline of COVID-19's business last year. The company has made significant investments in the treatment of anti-cancer drugs and other diseases to help 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 aimed at the genetic source or cells of patients to cure or significantly alter the progression of the disease. Some health experts predict that these therapies will replace traditional lifelong treatments that people use to manage chronic diseases.
Beqvez will compete with Hemgenix from CSL Behring in Australia, the first FDA approved gene therapy for hemophilia B, which will be launched in 2022 at a price of $3.5 million per dose.
It is worth noting that some health experts have stated that high costs and logistics issues have limited the adoption of Hemgenix and another approved gene therapy for hemophilia A.