17/09/2026 Phase III CELESTIMO study met its primary endpoint of PFS, with Lunsumio + lenalidomid in Follicular Lymphoma OS Therapies announces FDA Alignment on Phase 3 Design and Project Orbis Participation for OST-HER2 in metastatic osteosarcoma US FDA Approves Label Update for IBTROZI (taletrectinib), with Updated Duration of Response in TKI-Naïve Advanced ROS1-Positive NSCLC NICE Recommends Enhertu for HER2-Low Advanced Breast Cancer Phase III CELESTIMO study met its prim

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.S.

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 Regula

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