Its price is $ 3.5 million … the most expensive drug in the world, a new hope for “hemophilia” patients

Its price is $ 3.5 million … the most expensive drug in the world, a new hope for “hemophilia” patients
Its price is $ 3.5 million … the most expensive drug in the world, a new hope for “hemophilia” patients

Its price is $ 3.5 million … the most expensive drug in the world, a new hope for “hemophilia” patients

وافقت هيئة الغذاء والدواء الأميركية على علاج جيني لمرض «الهيموفيليا بي»، ما يُعرف بمرض «الناعور» من شركة «سي إس إل بيهرينغ»، وهو جرعة تُعطى لمرة واحدة لشفاء المرضى من العلاجات المنتظمة، لكنه يكلف 3.5 مليون دولار، مما يجعله أغلى دواء in the world.

Hemophilia is a rare condition that causes the blood to not clot normally due to a lack of the proteins needed for clotting, and the loss of these proteins can cause long-lasting bleeding after an injury.
People with this disease are prohibited from playing sports that could injure them. More than one research team around the world has presented genetic treatments for the disease, but these treatments, which are still being tested, offered no hope of stopping patients from injecting themselves regularly once or twice a week.
A large study found that the treatment, called Hemaginex, reduced the number of expected bleeding events by 54% over a year 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 diseases with serious consequences by addressing their underlying causes.
Peter Marks, director of the US Food and Drug Administration’s Center for Biological Evaluation and Research, says that despite advances in the treatment of hemophilia, measures to prevent and treat bleeding can affect patients’ lifestyles. He said that Hemaginex represents an important advance in the development of innovative treatments for people affected by the disease.
Traditional treatment for hemophilia works by transporting missing proteins called clotting factors, which the body needs to form blood 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. Clotting Factor IX is also known as “Christmas Factor” because it was discovered in 1952 in a child named “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 care system.
Bloomberg stated that approximately 16 million people in the United States and Europe have hemophilia B, while hemophilia A is more common.

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