Rs 28.7 crore for a single dose; Hemgenix is world’s most expensive medicine
'Hemgenix' treats haemophilia, an inherited bleeding disorder
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FollowUnited States regulators some days ago approved 'Hemgenix', a one-dose gene therapy medicine to treat haemophilia that is hogging the headlines for a different reason -- it is the world's most expensive medicine. It is priced at 35 lakh Dollars or Rs 28.7 crore.
Haemophilia is an inherited bleeding disorder in which the blood does not clot properly. This problem can cause spontaneous bleeding or uncontrolled bleeding injuries, or surgical procedures. The new gene therapy drug is developed by CSL Behring.
"Although the price is a little higher than expected, I think it has the potential to be successful because existing drugs are too expensive and haemophilia patients are afraid of constant bleeding," said Brad Lonkar, a biotechnology investor and CEO of Loncar Investments.
So, what causes hemophilia?
According to the Centers for Disease Control and Prevention (CDCP), 'Blood contains many proteins called clotting factors that can help to stop bleeding. People with haemophilia have low levels of either factor VIII (8) or factor IX (9). The severity of a person's haemophilia is determined by the number of factors in the blood. The lower the amount of the factor, the more likely it is that bleeding will occur, which can lead to serious health problems.'
Since haemophilia is a relatively uncommon condition, there has not been much interest in developing a comprehensive drug such as this condition. As per the CDCP, 'the best way to treat haemophilia is to replace the missing blood clotting factor so that the blood can clot properly. This is typically done by injecting treatment products, called clotting factor concentrates, into a person's vein.'
The new drug 'Hemgenix' has to be given only once. The clinical study showed that this therapy reduced the number of expected bleeding events by 54 per cent over the course of one year. With this therapy, 94 per cent of patients did not need to get expensive injections like 'Factor IX' again and again.
Over the years, though gene therapies have come to improve the health conditions of many diseases, including Thalassemia, by fixing their underlying causes, pricing has been the major issue in the treatment.
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