Good news for children with MCT8 deficiency, a rare genetic thyroid disease. The European Medicines Agency (EMA) has approved the drug tiratricol, rediscovered and investigated by Erasmus MC for this patient group. This approval means that the drug will soon be available to patients throughout Europe.
The approval is a huge breakthrough as it is generally rare for the EMA to approve a drug that has been examined and proven effective on the initiative of physician-scientists. MCT8 deficiency, also known as Allan-Herndon-Dudley Syndrome, affects only about 600 patients worldwide, with many children not surviving past the age of 18.
TTO played an essential role in setting up a collaboration between the researchers at Erasmus MC and Egetis Therapeutics, a Swedish pharmaceutical company. TTO also facilitated the licensing arrangements that support the research efforts at Erasmus MC.
This collaboration is crucial in advancing the development and approval of tiratricol.
With approval from the European Medicines Agency (EMA), Egetis Therapeutics can now begin producing the medicine for the European market.
TTO's contribution to this process underscores the importance of academic institutions working closely with pharmaceutical companies aiming to bring the innovative treatments to the market. This collaboration demonstrates a powerful synergy that can be achieved between academic institutions and pharmaceutical companies. It proves that combining scientific research with industry expertise can lead to innovative treatments reaching the market and creating valuable impact.
Read the full article in Amazing Erasmus MC
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