On January 31, 2025, expert clinicians, researchers, and global advocacy leaders convened in Amsterdam, the Netherlands, to meet with the European Medicines Agency (EMA) and EORTC, exploring strategies to develop new treatments for ultra-rare sarcomas as a model for ultra-rare tumors. The discussion focused on lessons learned from efforts to secure regulatory approval for sirolimus for the treatment of EHE in the European Union, highlighting progress and ongoing challenges in developing drugs for ultra-rare diseases.

The EHE Foundation, alongside EHE Rare Cancer Charity UK and EHE ITALIA Associazione Non Solo LAURA ODV, plays an active role in ensuring regulatory bodies understand the urgent unmet needs of people living with EHE. Together, we support critical research that not only generates data for regulatory review but also helps inform treatment practices for EHE patients worldwide.