TREAT-AHDS
| Reference number | |
| Coordinator | Rare Thyroid Therapeutics International AB |
| Funding from Vinnova | SEK 5 000 000 |
| Project duration | September 2017 - March 2022 |
| Status | Completed |
| Venture | Eurostars |
Purpose and goal
The project´s objective was to develop and advance towards market the first drug for the treatment of MCT8 deficiency, also called Allan Herndon Dudley Syndrome, a rare condition of thyroid hormone signaling that affects 1 / 70,000 men. An adapted formulation and analysis method have been developed, a patient registry has been established and a clinical study in very young children is ongoing and will report results in 2024. Interactions with regulatory authorities have been carried out and preparations for a marketing authorisation application which is expected to be filed in 2023.
Expected results and effects
The project has gone well according to plan and the results have continued to be very promising and interactions with regulatory authorities have been positive. The company feels confident that the project will result in the first approved drug for the treatment of AHDS on the market, which we expect to launch with our own commercial organization in 2024.
Planned approach and implementation
The project has been run as a collaboration with Erasmus Medical Center, Rotterdam, the Netherlands. In addition, a number of consultants, experts and subcontractors have been involved in the project for the various sub-projects. Regular meetings have been held to follow the project development in the various sub-projects. Cooperation and progress of the project has worked well, the delays that have occurred in comparison with the original plan are dependent on external factors.