FDA has granted orphan drug designation to RedHill Biopharma Ltd opaganib, a novel, host-directed, orally administered small molecule drug, for treating neuroblastoma, a rare and aggressive pediatric cancer.
In addition to ongoing clinical trials, RedHill is also conducting a Phase 2 study to investigate Opaganib’s potential to improve treatment outcomes for patients with certain types of breast and prostate cancer.
Dr. Mark Levitt, Chief Scientific Officer at RedHill, acknowledged the significant milestone of receiving a second FDA orphan-drug designation for opaganib in oncology, this time for neuroblastoma.
Following the earlier designation for cholangiocarcinoma (CCA) in phase 2a study, he emphasized that this recognition highlights opaganib’s broad potential in oncology.
Dr. Levitt also noted promising data in solid tumors like prostate cancer, neuroblastoma, and CCA and its potential role in enhancing hormone receptor pathway therapies, which will be explored in an upcoming Phase 2 study.
Opaganib is currently being evaluated in multiple clinical trials for various oncology, viral, inflammatory, and diabetes and obesity-related indications, including COVID-19, Ebola, acute respiratory distress syndrome (ARDS), and radio/chemical protection.
The ODD provided opaganib with 7 years of marketing exclusivity and potential benefits like accelerated development, grant funding, and tax credits.
The company is collaborating with various U.S. government agencies to advance the development of opaganib for neuroblastoma and other promising drug candidates.
Source: RedHill’s Opaganib Granted Orphan Drug Designation by the FDA for Childhood Cancer, Neuroblastoma
https://www.prnewswire.com/news-releases/redhills-opaganib-granted-orphan-drug-designation-by-the-fda-for-childhood-cancer-neuroblastoma-302230324.html
