So far, people who suffer from the so-called hemophilia have received regular infusions. A new drug from Australian drug manufacturer CLS Ltd makes this procedure unnecessary. But the new therapy method has its price.
So far, an injection of the world’s most expensive drug has cost $2.1 million. The drug Zolgensma from the pharmaceutical giant Novartis is used to treat spinal muscular atrophy (SMA), a rare hereditary disease that usually leads to premature death if left untreated. This record amount has now been surpassed: The Australian drug manufacturer CLS Ltd is demanding around 3.4 million dollars for its drug Hemgenix against the bleeding disease hemophilia B – for a single dose. According to the company, the drug only has to be administered once and reduces expected bleeding over the course of a year by 54 percent.
“Although the price is a bit higher than expected, I believe it has a chance of being successful,” Bloomberg quoted Brad Loncar, a biotechnology investor. On the one hand, the existing drugs are also very expensive, on the other hand, hemophiliac patients live in constant fear of bleeding. The drug Aduhelm from Biogen Inc. for Alzheimer’s in the USA recently showed that pricing, especially for new drugs, is a sensitive issue: the high costs contributed to the drug flopping.
According to Peter Marks, director at the US Center for Biological Evaluation and Research, there are already advances in the treatment of hemophilia. According to him, the measures required to prevent and treat bleeding affect the patient’s quality of life. Hemgenix represents an important advance in the development of innovative therapies for people affected by the disease, he is quoted as saying by “Bloomberg”.
The missing proteins, so-called coagulation factors, which the body needs to form blood clots and stop bleeding, have been given to hemophilia patients by infusion. Instead, in Hemgenix, a gene that can make the missing clotting factors is introduced into the liver, where it starts making the protein responsible for it.
