RedHill Biopharma Receives FDA Rare Pediatric Disease Designation for Opaganib for Neuroblastoma

RedHill Biopharma announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease (RPD) designation to its drug, opaganib, for the treatment of neuroblastoma. This designation is in addition to the existing orphan drug designation for neuroblastoma. The RPD designation provides potential eligibility for a Priority Review Voucher (PRV) and, along with the orphan drug designation, may offer benefits such as accelerated development and review times, application fee waivers, tax credits, and seven years of marketing exclusivity if opaganib is approved. Recent preclinical data presented at the 2026 American Association for Cancer Research (AACR) Annual Meeting showed positive effects of opaganib as a potential add-on therapy in models of neuroblastoma and triple-negative breast cancer. The neuroblastoma market is projected to reach approximately $3.5 billion by 2032. Opaganib is an investigational, oral, small molecule drug targeting multiple indications, including oncology and viral diseases.