General Updates | Key trials of interest in the thalassemia space

Well, of course, the mitapivat trials are ongoing, and this will give us the chance to find out about long-term efficacy and safety data. Data so far are encouraging, but of course we need the long-term results. And mentioning this, Mitapivat got a positive opinion from the EMA, from the committee of EMA, just a few days ago, and it will probably get an approval in Europe for the treatment of both TDT and NTDT thalassemia. And, of course, it’s exciting to have an oral new agent for the treatment of anemia in these patients. 

Then there is an ongoing trial with a new chelating agent, currently under the name of SP-420. This agent has a very favorable dosing schedule, so it can be given orally only three times a week, and we really wait for the results of this trial to see if it is possible to find a new treatment that would be better for the quality of life of patients with a schedule that is favorable compared to the everyday schedule of all chelating treatments. 

There is also an antibody for the iron overload of NTDT that has recently initiated the clinical trial. And of course, there are multiple gene therapy trials, either with the gene editing technique or the gene editing technique, including also base editing technology, which is very exciting, so apart from the agents that have already been approved for gene therapy. So overall, I think it’s a very exciting time for thalassemia as we get new treatments introduced for our patients.

This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.