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 Aminex,

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 Designation f

02/10/2025 Aminex Therapeutics' AMXT 1501 + DFMO received the FDA Orphan Drug Designation for Neuroblastoma (Ref) The US FDA granted the Orphan Drug Designation to Aminex Therapeutics' AMXT 1501 (polyamine transport inhibitor) + difluoromethylornithine for the treatment of patients with neuroblastoma. Mark Burns, CSO and President, Aminex Therapeutics: "Receiving Orphan Drug Designation for AMXT 1501 in combination with DFMO represents an important milestone in our mission to

06/08/2025 Jazz Pharmaceuticals' Modeyso received accelerated approval from the FDA for Recurrent H3 K27M-mutant Diffuse Midline Glioma (Ref) The US FDA granted accelerated approval to Jazz Pharmaceuticals' Modeyso™ (dordaviprone; oral antagonist of dopamine receptor D2 and agonist of ClpP) for the treatment of adult and pediatric patients 1 year of age and older with diffuse midline glioma harboring an H3 K27M mutation with progressive disease following prior therapy. Contin