there Federal Drug Administration (FDA) recently approved a new treatment for a rare blood clotting disorder: hemophilia B. proper blood clotting. The most common symptoms include spontaneous and recurrent bleeding.
The drug is called hemigenix It is the most expensive drug in the world; Each dose, in fact, It cost $3.5 million. According to a recent analysis, the price is relatively “fair” to the results achieved by the treatment. Hemophilia B affects more men than women, with an estimated 8,000 male patients in the United States.
The treatment currently used gives the patient a protein needed for clotting; However, its effectiveness decreases over time. Universe Hemophilia B is a lifelong diseaseOrdinary drug use is a luxury for the few. Experts estimate that treatment for one person is between $21 and $23 million (in Europe, treatments are cheaper, but still upwards of $10 million).
A separate case, however, it is hemigenix: It should only be taken once intravenously at a modest $3.5 million. The drug exploits a viral vector, which transmits to the patient’s DNA the genetic information that codes for the synthesis of a clotting protein, that is, the so-called Factor IX.
Currently, hemigenix It was only tested in two specific studies and the results were more than positive; Indeed, patients suffered from a Increase activity levels Factor IX. The side effects may seem mild in nature, in fact headaches and flu symptoms are among them. However, there was an unusual increase in enzymes in the liver which needs to be monitored by doctors in the future.
To stay on this topic, another exceptional drug was recently approved, capable of delaying the onset of type 1 diabetes.