FDA Grants RPDD to RedHill Biopharma for Neuroblastoma Therapy

Rare Daily Staff

The U.S. Food and Drug Administration has granted RedHill Biopharma rare pediatric disease designation to its experimental therapy opaganib for the treatment of neuroblastoma, a high-risk childhood cancer.

The designation builds on opaganib’s existing orphan drug status in neuroblastoma and positions RedHill to potentially receive a Priority Review Voucher if the therapy is ultimately approved. The combined incentives tied to these designations may include expedited regulatory review, tax credits, user fee waivers, and up to seven years of market exclusivity.

Neuroblastoma remains one of the most challenging pediatric cancers, accounting for roughly 15 percent of childhood cancer-related deaths in the United States. While it is the most common cancer diagnosed in infants, outcomes remain poor for high-risk patients, who represent about half of cases and face five-year survival rates of approximately 50 percent.

Opaganib is a first-in-class, orally administered sphingosine kinase-2 inhibitor designed to act through a host-directed mechanism. Beyond oncology, the small molecule is being explored across a range of indications, including viral, inflammatory, and metabolic diseases.

RedHill’s growing focus on the indication is supported by preclinical data presented earlier this year at the American Association for Cancer Research annual meeting. In those studies, opaganib demonstrated potential as an adjunct to chemotherapy, with evidence suggesting it may destabilize the oncogenic driver n-Myc. The mechanism appears to involve increased ceramide production and the induction of cancer cell death, pointing to a possible role in overcoming treatment resistance.

RedHill Chief Scientific Officer Mark Levitt said the findings reinforce confidence in opaganib’s potential to improve outcomes, particularly in high-risk neuroblastoma. The company is continuing development discussions with academic partners, including Penn State University and the Beat Childhood Cancer consortium.

RedHill is also advancing opaganib in a phase 2 study in combination with Bayer’s darolutamide for metastatic castration-resistant prostate cancer.