19/05/2026 Cullinan’s CLN‑049 earns FDA Orphan Drug Designation in relapsed/refractory AML FL118 Receives Dual FDA Designations for Osteosarcoma, Advancing Rare Pediatric Cancer Treatment Cullinan’s CLN‑049 earns FDA Orphan Drug Designation in relapsed/refractory AML (Ref) LN-049, a novel FLT3 x CD3 T cell engager, has received Orphan Drug Designation for relapsed/refractory acute myeloid leukemia (AML) CLN-049 is in Phase 1 trials, including multiple ascending dose studies i

14/04/2026 FDA Grants Fast Track to Daretabart for High‑Risk Neuroblastoma FDA sBLA Submission & NCCN Guideline Inclusion for BIZENGRI in NRG1 Fusion–Positive Cholangiocarcinoma Obsidian Therapeutics and Galera Therapeutics Announce Merger Agreement to advance OBX-115 TIL Therapy FDA Grants Fast Track to Daretabart for High‑Risk Neuroblastoma ( Ref ) Renaissance Pharma's Daretabart (hu1418K322A), a novel anti-GD2 monoclonal antibody, has received Fast Track status from the U.

08/04/2026 FDA Grants Orphan Drug Status to Plus Therapeutics’ REYOBIQ™ for Pediatric Malignant Gliomas FDA Grants Orphan Drug Designation to Compass Therapeutic’s Tovecimig for Biliary Tract Cancer FDA Grants Orphan Drug Status to Plus Therapeutics’ REYOBIQ™ for Pediatric Malignant Gliomas ( Ref ) Plus Therapeutics’ REYOBIQ™ (rhenium Re186 obisbemeda) has been granted orphan drug status for pediatric malignant gliomas, with scope expanded to include pediatric ependymoma Regu

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