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

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 [1], [2] 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 [3] 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 TEL AVIV, Israel and RALEIGH, N.C. , Aug.

26, 2024 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL ) ("RedHill" or the "Company"), a specialty biopharmaceutical company, 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. Orphan Drug designation provides for a seven-year marketing exclusivity period should opaganib be approved in neuroblastoma and may confer addition.