Approved the most expensive drug in the world. The go came from the Food and drug administration (Fda), a US regulatory body. It is about theHemgenix, a medicine to treat haemophilia B, a rare genetic disease.
Hemgenix, the therapeutic cure costs 3.5 million dollars per dose
The new gene therapy is manufactured by the pharmaceutical company CSL Behring. It will cost $3.5 million per dose, which, as Bloomberg reports, makes Hemgenix the most expensive drug out there.
The clinical studies carried out to date have found that Hemgenix would be able to eliminate, in over half of the patients tested, the bleeding cases associated with the disease, with potential savings on healthcare costs.
In Europe, the medicine is still being evaluated. It will be the European Medicines Agency (EMA) that will have to pronounce.
Hemophilia B: symptoms and how it has been treated so far
Hemophilia B is classified as a rare genetic disease caused by the absence of a blood clotting factor, which is a protein needed to stop bleeding when wounds occur.
This disease is estimated to affect 1 in 30,000 people in the global population. The pathology is related to the sex chromosome X and that is why the vast majority of affected individuals are men. Women tend to be healthy carriers. Despite this, data in hand, 10-25% of female carriers show mild symptoms. Rare cases in which moderate or severe symptoms emerge.
Symptoms include:
- episodes of prolonged or heavy bleeding following wounds, trauma and/or surgery;
- in the most serious cases, bleeding can also occur spontaneously, without a clear cause; these episodes can lead to serious complications, such as bleeding into joints, muscles or internal organs.
Until now, the disease has been treated by administering the missing clotting factor as a preventive measure, to avoid cases of bleeding or from time to time to stop any bleeding.
In the most severe episodes, treatment with intravenous infusions. This treatment is very expensive and over time it can decrease in effectiveness.
Hemgenix, how it acts on the disease
Hemgenix works differently than the treatments used up to now: is an adenoviral vector administered intravenously in a single dose and within it carries the gene for the missing clotting factor. Once in the circulation, the gene is expressed in the liver, where it makes the protein to prevent bleeding episodes.
Hemgenix, as explained by Repubblica, was tested in two clinical studies and on 57 men with severe or moderately severe haemophilia B. In one of the studies, participants experienced increased activity levels of the missing clotting factor (resulting in less need for preventative treatment) and a 54% reduction in the rate of annual bleeding events.
The most common adverse reactions associated with the drug included increased liver enzymes, headache, mild intravenous infusion reactions, and flu-like symptoms.
Photo source: ANSA