RedHill’s SPHK2 inhibitor opaganib wins FDA rare pediatric disease designation for neuroblastoma.

Edited by Robert McTiernan··2 min read

RedHill Biopharma Ltd. (Nasdaq: RDHL) announced receipt of FDA rare pediatric disease (RPD) designation for opaganib (ABC294640) in neuroblastoma, adding a second federal designation to the asset in the same indication and unlocking eligibility for a Priority Review Voucher (PRV) upon approval, subject to certain conditions.

PRVs have traded at between USD 100 million and USD 205 million in recent disclosed transactions, providing a potential non-dilutive capital mechanism for a company with a cash balance of USD 4.1 million as of December 31, 2025. Combined with the orphan drug designation RedHill received for opaganib in neuroblastoma in August 2024, the asset now carries two overlapping federal designations for the same indication.

The designation may provide development incentives including tax credits, user-fee exemptions, and, if ultimately approved for the indication, seven years of US orphan exclusivity.

Opaganib is a first-in-class, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor originally discovered at the Medical University of South Carolina and licensed by RedHill from Apogee Biotechnology Corporation in 2015. Its proposed mechanism in neuroblastoma centers on simultaneous inhibition of three sphingolipid-metabolizing enzymes — SPHK2, DES1, and GCS — which RedHill’s collaborators at Penn State University reported increases ceramide production, destabilizing n-Myc, a key oncogenic driver of high-risk neuroblastoma (HRNB). Preclinical data presented at the 2026 American Association for Cancer Research (AACR) Annual Meeting indicated that opaganib may enhance the efficacy of the oxaliplatin plus doxorubicin chemotherapy combination in HRNB models, with apoptosis induction as the proposed effector mechanism.

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No clinical trial in neuroblastoma has been initiated. RedHill has stated that further development discussions are ongoing with Penn State University and the Beat Childhood Cancer consortium. Opaganib has demonstrated a safety and tolerability profile across more than 470 participants in multiple clinical studies, including a Phase II/III study in hospitalized COVID-19 patients, but no efficacy data in neuroblastoma patients have been reported. As noted in RedHill’s full-year 2025 financial results, the neuroblastoma program remains at a pre-IND stage, dependent on external academic and consortium partnerships to advance.

Neuroblastoma accounts for approximately 10% of childhood cancers and 15% of pediatric cancer-related deaths in the US, with around 750 new cases diagnosed annually. Approximately half of all patients have high-risk disease, which carries a five-year overall survival rate of approximately 50% despite intensive multimodal therapy. The neuroblastoma market is projected to reach approximately USD 3.5 billion by 2032, according to Evaluate Ltd estimates cited by RedHill.

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