There Federal Drug Administration (FDA) has recently approved a new treatment for hemophilia B, a rare blood clotting disorder. proper blood coagulation. The most common symptoms include spontaneous and recurrent bleeding.
called medicine hemgenix and it is the most expensive drug in the world; In fact, every dose It cost $3.5 million. According to a recent analysis, the price is relatively “fair” for the results the treatment provides. Hemophilia B affects more men than women, with an estimated 8,000 male patients in the United States.
The currently used drug gives the patient a protein necessary for coagulation; however, its effectiveness decreases over time. Be hemophilia B is a lifelong disease, habitual drug intake is a luxury for few people. Experts estimate that treatment for a single person costs between $21 and $23 million (treatments are cheaper in Europe, but still higher than the equivalent of $10 million).
However, situation aside, hemgenix: should only be taken once intravenously, for a modest price of $3.5 million. The drug utilizes a viral-based vector that transmits genetic information encoding the synthesis of the coagulation protein, the so-called protein, into the patient’s DNA. Factor IX.
At the moment, hemgenix tested in only two specific studies and results are more than positive; In fact, patients are increased activity levels Factor IX. Side effects may seem mild in nature, in fact, they include headaches and flu symptoms. However, there was an unusual increase in enzymes in the liver that should be monitored by doctors in the future.
Without departing from the topic, an extraordinary drug that can delay the onset of type 1 diabetes has been approved more recently.
Source: Every Eye