RedHill Biopharma has received FDA rare pediatric disease designation for its investigational agent opaganib for the treatment of neuroblastoma, adding to the drug’s existing orphan drug designation in the indication. The RPD designation, which applies to diseases primarily affecting babies and young children, provides eligibility for a Priority Review Voucher, subject to certain conditions, while the orphan drug status may enable seven years’ marketing exclusivity upon approval, along with potential benefits including accelerated development and review timelines, PDUFA fee waivers and tax credits. The neuroblastoma market is projected to reach approximately $3.5 billion in 2032.
RedHill reported new preclinical data presented at the 2026 American Association for Cancer Research Annual Meeting showing positive effects of opaganib as a potential add-on therapy in models of neuroblastoma and triple-negative breast cancer. In high-risk neuroblastoma models, opaganib, as an add-on to chemotherapy, demonstrated the ability to destabilize the oncogenic driver n-Myc through increased ceramide production, enhancing programmed cancer cell death. Chief Scientific Officer Dr. Mark Levitt said the RPD designation, combined with supportive preclinical data and the drug’s clinically demonstrated safety and tolerability profile, reinforces the company’s belief that opaganib could improve outcomes in pediatric neuroblastoma, and noted that RedHill aims to advance development in collaboration with Penn State University and the Beat Childhood Cancer consortium.
Opaganib is described as a novel, potentially broad-acting oral small molecule sphingosine kinase-2 selective inhibitor with established safety and efficacy profiles. It is in development across multiple oncology indications, as well as viral, inflammatory, and diabetes and obesity-related conditions, including Ebola virus disease.
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