A new drug for the treatment of hemophilia, which may become the most expensive drug in the world, has not yet been released on the market. By message developer, its cost was estimated at three million dollars.
The company received an order from US regulators to wait until the end of 2021 to confirm the duration of the therapy’s effectiveness.
For a long time, it was believed that genetically transmitted diseases were generally incurable. So, in hemophilia, human cells do not produce enough of one of the blood coagulation proteins, so the patient needs to regularly inject the missing protein, which requires several hundred thousand dollars annually.
An effective solution to the problem was found recently – with the help of gene therapy. A neutralized virus is introduced into the patient’s blood, which gives the cell a missing copy of a healthy gene. This treatment is already approved for several conditions, such as those associated with hereditary blindness.
But a gene therapy drug is the most expensive drug in the world. The development of Biomarin Pharmaceutical, which, judging by successful clinical trials, is aimed at completely eliminating hemophilia, was estimated at almost three million dollars.
When the drug was injected, the protein concentration in the patient’s blood gradually dropped, while remaining at the end of the tests within the normal range. This raised concerns about how long the effect of the drug is.
Now the developer is continuing the third phase of testing the drug, the final results will be known in November next year. In this regard, the company was denied registration of the drug and was asked to wait for the results of the last stage of its testing.
– .
