RedHill’s Opaganib Granted Orphan Drug Designation by the FDA for Childhood Cancer, Neuroblastoma

RedHill Biopharma (Nasdaq: RDHL), today announced that the U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to opaganib for treatment of neuroblastoma, a type of childhood cancer that develops from immature nerve cells and accounts for 15% of all pediatric cancer-related deaths.

·             Neuroblastoma is rare but is the most common infancy malignancy with a median age of diagnosis of 17 months. In the U.S., it accounts for up to around 10% of all childhood cancer cases and 15% of pediatric cancer-related deaths

·             Orphan Drug designation provides for seven-years’ marketing exclusivity should opaganib be approved in neuroblastoma and may confer additional benefits such as accelerated development and review times, potential grant funding and possible tax credits

·             The neuroblastoma market is expected to reach almost $1.5 billion before mid-2030s 

·             This is the second orphan drug designation by the FDA for opaganib in oncology, after cholangiocarcinoma (bile duct cancer, CCA)

·             With multiple U.S. government collaborations ongoing, opaganib is a novel, host-directed, potentially broad acting, orally administered small molecule drug with demonstrated safety & efficacy profiles

“RedHill is proud to have received a second orphan-drug designation for opaganib in oncology, following its previous designation for cholangiocarcinoma (CCA, also known as bile duct cancer). This designation for neuroblastoma – the most common infancy malignancy and for which new options are urgently needed – adds to opaganib’s potential as a novel oncological agent,” said Dr. Mark Levitt, Chief Scientific Officer at RedHill. “Opaganib has broad oncology potential with promising preliminary clinical data in solid tumor cancers such as prostate cancer and CCA, and data from a range of U.S. government supported and Apogee conducted preclinical studies in various indications, including radioprotection, and also in combination with RedHill’s RHB-107. We also see such utility extending to the potential for opaganib to have a sensitizing effect in hormone receptor pathway inhibition therapy, which the Company expects to test in a planned externally funded Phase 2 study.”