“Golden” pill. The United States approved the most expensive drug in the world – one dose costs $ 3.5 million

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Analysis of the cost-effectiveness of the drug shows that it's worth every penny.

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Recently, the US Federal Drug Administration (FDA) approved a new drug for the treatment of a rare genetic bleeding disorder. The price of the drug is so high that it has literally become the most expensive drug in the world, writes Science Alert.

At first glance, it seems that $ 3.5 million for just one dose of the drug is too expensive, but a recent analysis of the economic effectiveness of the drug shows that this is a very fair price for what the drug achieves. At least in the USA.

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The new drug is called Hemgenix and is essentially a gene therapy for the treatment of hemophilia B, a rare genetic disease that causes decreased clotting blood. One of the most common symptoms of the disease is spontaneous and repeated bleeding, which is extremely difficult to stop.

The researchers note that hemophilia B is more common in men, but it is difficult to name the exact number of people suffering from the disease – according to preliminary estimates, about 8,000 thousand men suffer from this rare disease in the United States alone.

There is currently a drug used to treat this rare genetic disease, but patients need to take it for life and it is also very expensive. According to preliminary estimates, it will take about 21-23 million dollars to have access to such therapy. However, doctors warn that over time, the effectiveness of treatment may decrease. In the UK, the cost of treatment today is lower than in the US and other European countries, but it is still tens of millions of dollars.

Hemgenix is ​​a single-dose intravenous drug, which in fact represents only a small fraction of the cost treatment that exists today. The product is transferred into the patient's body using a virus-based vector that has been specifically designed to deliver DNA to target cells in the liver. The information is then replicated by the cells and propagates the instructions for a blood clotting protein (factor IX).

Note that Hemgenix has undergone two efficacy and safety studies. The results indicate that slightly more than fifty study participants suffering from moderately severe hemophilia had significantly increased levels of factor IX activity. In addition, according to the results of the study, the number of uncontrolled bleeding in recipients was halved.