Cellectar Biosciences receives rare pediatric disease designation from U.S. FDA for Iopofosine I 131 in relapsed or refractory pediatric high-grade glioma

CAR-T Digest - August 2020

Weekly Digest - October 2025

27 October 2025: Cellectar Biosciences receives rare pediatric disease designation from U.S. FDA for Iopofosine I 131 in relapsed or refractory pediatric high-grade glioma

Cellectar Biosciences received Rare Pediatric Disease Designation (RPDD) from the FDA for Iopofosine I 131 in relapsed or refractory pediatric high-grade glioma (r/r pHGG), following prior orphan drug designation
The CLOVER-2 Phase 1b trial showed notable efficacy with average progression-free survival (PFS) of 5.4 months and overall survival (OS) of 8.6 months, extending beyond typical outcomes in this aggressive cancer type
Higher dosing cycles (≥4 infusions) demonstrated enhanced outcomes with average PFS of 8.1 months and OS of 11.5 months, including objective responses in two patients, underscoring the potential of iopofosine I 131 as a targeted radiotherapeutic
Safety data revealed no significant off-target toxicity, with adverse events limited to manageable hematologic effects—supporting iopofosine I 131’s favorable safety and tolerability profile
Cellectar may qualify for a Priority Review Voucher (PRV) upon NDA approval, positioning the company for strategic partnerships and accelerated advancement of a potential first-in-class precision radiotherapy for pediatric brain tumors