12/03/2026 Aminex Therapeutics' AMXT 1501 received the FDA ODD for Glioma Aminex Therapeutics' AMXT 1501 received the FDA ODD for Glioma ( Ref ) The US FDA granted the orphan drug designation to Aminex Therapeutics' AMXT 1501 (polyamine transport inhibitor) + difluoromethylornithine for the treatment of malignant glioma, including diffuse intrinsic pontine glioma (DIPG). The Beat Childhood Cancer Research Consortium at Penn State College of Medicine, in partnership with Amine

27/10/2025 Cellectar Biosciences' iopofosine I131 received the rare pediatric drug designation for pediatric high-grade glioma ( Ref ) The US FDA granted the rare pediatric drug designation (RPDD) to Cellectar Biosciences' iopofosine I131 in inoperable R/R pediatric high-grade glioma (pHGG). The FDA previously granted Orphan Drug Designation for iopofosine I 131 for the treatment of pHGG James Caruso, President and CEO, Cellectar: “ Receiving Rare Pediatric Disease Designatio

07/10/2025 Precision NeuroMed's cinetredekin besudotox received the FDA orphan drug designation for GBM ( Ref ) The US FDA granted the...

08/09/2025 Beyond Air's BA-101 received the Orphan Drug Designation for GBM ( Ref ) The US FDA granted the Orphan Drug Designation to...