Pharmaceutical giant Pfizer announced today that it has received approval in the United States for a new gene therapy against hemophilia, a rare genetic disease that causes massive and spontaneous bleeding.
The treatment, called Beqvez, targets hemophilia B and is given in a single dose.
Hemophilia is caused by an anomaly in the X chromosome gene, and there is a protein deficiency in the body called the coagulation factor (Factor VIII for hemophilia A and factor IX for hemophilia B). It mainly affects men.
The classic treatment consists of transfusions, which introduce the missing factor several times a week.
"A single treatment with Bekvez could change that," said Adam Zucker, an official in the hemophilia program at the University of Pennsylvania, whose words are quoted in a Pfizer statement.
The treatment works by using a virus modified so that it is not dangerous, in order to introduce a functional copy of the factor IX gene, which the patient's body can then produce on its own.
One dose of the treatment in the US will cost $3,5 million, according to Pfizer, which adds that current treatments can cost between $600.000 and $1,1 million per person per year.
The price a patient would pay for Bekvez would depend on how much is covered by their health insurance.
According to Pfizer, in many cases patients would not pay full price.
The treatment has already been approved in Canada and is now under review by the European Medicines Agency (EMA).
Gene therapy against hemophilia A, more common than hemophilia B, has already been approved in the US in 2023.
Bonus video:
