News & Events
New Therapies Against Rare Blood Diseases Approved
- February 24, 2020
- Posted by: Wiley M. Wagner
- Category: Science
Today, it was reported that the U.S. Federal Food and Drug Administration has officially included elzonris in the list of approved medicinal compounds – or rather, Elzonris liquid solution, which has shown promising character in the treatment of one of the rarest and most serious blood diseases. We are talking about blast plasmacytoid dendritic neoplasm, which is one of the most severe forms of leukemia, which is especially susceptible to children from two years of age with the corresponding genetic heritage. The drug was previously tested on the final group of patients, consisting of adults and children from two years old – the results were really impressive and unusual.
Researchers carefully studied the effect of Elzonris solution on patients suffering from this form of leukemia – while in the context of the study, two groups of patients were composed, one of which was a group of patients with remission, and the second with relapse. After some time, scientists found that 54% of the first group of patients treated with this substance showed complete remission, either with inactive side effects or without them at all.
In the second group, elzonris had a remission effect in two patients, with approximately the same side parameters. Such an amazing and without a doubt the resounding success of clinical trials of Elzonris allowed him to take an honorable place in the official list of drugs for leukemia, authorized by the US Federal Commission for the Quality of Food and Drug Administration. It is assumed that this substance will enter the US wide medical stream by the end of this month.
Leukemia in itself is a rather problematic disease that is not always amenable to treatment and remission even with the most favorable factors and outcomes – not to mention effectively combating it with the help of experimental therapies and substances. It is worth noting the fact that Elzonris will have to go through an additional stage of clinical testing.