Its price is $ 3.5 million ... the most expensive drug in the world, new hope for "hemophilia" patients

The US Food and Drug Administration has approved a gene therapy for hemophilia B, known as haemophilia, from CSL Behring. It is a one-time dose to cure patients from regular treatments, but it costs $3.5 million, making it the most expensive drug. In the world.

Hemophilia is a rare disorder that causes the blood not to clot normally due to a lack of proteins needed for clotting, and the loss of these proteins may cause continuous bleeding for long periods after any injury.

People with this disease are prohibited from practicing any sports that may cause them any injury.

More than one research team around the world presented genetic treatments for the disease, but these treatments, which are still under trial, did not give hope to rid the patient of injecting himself regularly once or twice a week.

A major study found that the treatment, called Hemaginex, reduced the number of expected bleeding events over the course of a year by 54%, and 94% of patients recovered from the time-consuming and expensive injections.

"The gene therapy product will be attractive to some," Brad Loncar, a biotechnology investor and CEO of Loncar, which invests in medical treatments, told Bloomberg.

And Bloomberg reported that gene therapies can dramatically improve a range of conditions with severe consequences by fixing their underlying causes.

In the context, Peter Marks, director of the US Food and Drug Administration's Center for Biological Evaluation and Research, says that despite progress in treating hemophilia, measures to prevent and treat bleeding can impair patients' lifestyle.

He said that Hemaginex represents an important advance in developing innovative treatments for people affected by the disease.

Conventional treatment for hemophilia works by transporting missing proteins, called clotting factors, that the body needs to form clots and stop bleeding.

But Hemaginex works by delivering a gene that can produce missing clotting factors in the liver.

Hemophilia B patients inject themselves with coagulation factor IX, a treatment they continue to take.

Coagulation factor IX is also known as the “Christmas factor” in relation to its discovery in 1952 in a child called “Stephen Christmas.” It is one of the proteins involved in the process of the coagulation system, and belongs to the peptidase family. The World Health Organization lists the compound for this factor as one of the essential medicines. that should be present in the basic health system.

Bloomberg indicated that about 16 million people in the United States and Europe have hemophilia B, while hemophilia A is more common.

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