The U.S. Food and Drug Administration (FDA) has granted orphan-drug designation to RedHill Biopharma’s 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.
 
Orphan Drug designation provides for a seven-year marketing exclusivity period 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.
 
“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.”
 
Neuroblastoma usually affects children aged five or younger but does also occur in older children. In the United States, approximately 650 new cases are diagnosed each year, accounting for around 10% of all childhood cancer cases.
 
Opaganib is in development for multiple oncology, viral, inflammatory and diabetes and obesity-related indications, including COVID-19, Ebola, acute respiratory distress syndrome (ARDS) and radio/chemical protection.