Hemgenix is gene therapy for adults with hemophilia B, an inherited bleeding disorder that causes people to not produce enough protein to form blood clots.
Hemgenix is gene therapy for adults with hemophilia B, an inherited bleeding disorder that causes people to not produce enough protein to form blood clots.

The world's medical progress has reached incredible levels, as people enter the age of miraculous cures, including the world's most expensive drug used to treat hemophilia.

The US has just approved the gene therapy for haemophilia B from biopharmaceutical company CSL Behring. At $3.5 million per dose, this is considered the most expensive drug in the world.

The company's research shows that with this therapy, patients need only one infusion without having to follow the usual course of treatment.

A single-use drug called Hemgenix from CSL Behring reduced patients' expected bleeding episodes by 54% over the course of a year by up to 54%. It also helps 94% of patients not need the time-consuming and expensive clotting factor IX transfusion today.

Brad Loncar, a biotech investor and chief executive officer of Loncar Investments, said: "Although the cost is higher than expected, I think the drug has a good chance of succeeding because firstly, the existing drugs are also very expensive, and second, patients with this disease live in constant fear. Therefore, gene therapy will be an option for some people."

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Peter Marks, director of the Food and Drug Administration's (FDA) Center for Biological Evaluation and Research, said that although there have been advances in the treatment of hemophilia, measures need to be taken. needed to prevent and treat bleeding disorders that may impair a patient's quality of life.

"Hemgenix represents an important advance in the development of innovative therapies specifically for patients with hemophilia," said Marks.

The traditional treatment for hemophilia is to infuse missing proteins, called clotting factors, to form clots and help stop bleeding. Meanwhile, Hemgenix works by introducing a gene into the liver that works to make the factor IX protein.

Gene therapy will be manufactured in Lexington, Massachusetts - USA by uniQure NV. This is the company that sold the rights to commercialize Hemgenix to CSL Behring in 2020.

Gene therapies can dramatically improve a wide range of diseases by correcting the underlying causes. Novartis AG's Zolgensma for infants with spinal muscular atrophy cost $2.1 million when approved in 2019, while Bluebird Bio Inc's Zynteglo. for the blood disorder beta thalassemia cost $2.8 million earlier this year.

Price is an issue for new drugs. High costs make drugs like the Alzheimer's drug Biogen Inc.'s Alzheimer's drug Aduhelm. (USA) and Bluebird's Zynteglo in Europe became bankruptcies.

According to statistics, one in 40,000 people has hemophilia B, mostly men. Patients are usually treated with continuous intravenous infusion to maintain adequate levels of missing clotting factors. However, with the new drug, the patient only needs one infusion.

According to uniQure, about 16 million people in the US and Europe have hemophilia B. Meanwhile, hemophilia A is more common, with five times as many patients.

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