RedHill's Opaganib Gets FDA Rare Pediatric Disease Designation

RedHill Biopharma Ltd. announced that its investigational drug, opaganib, has received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for the treatment of neuroblastoma. This new designation complements opaganib's existing Orphan Drug Designation for neuroblastoma, offering potential benefits such as a Priority Review Voucher, accelerated development and review times, FDA fee waivers, tax credits, and seven years of marketing exclusivity if approved. The company highlighted new preclinical data presented at the 2026 AACR Annual Meeting, which showed positive effects of opaganib as a potential add-on therapy in models of neuroblastoma and triple-negative breast cancer, with the neuroblastoma market projected to reach $3.5 billion by 2032.