NeuroNOS Granted FDA Orphan Drug Designation for Glioblastoma, the Most Common and Deadliest Primary Malignant Brain Cancer in Adults

Core Insights - NeuroNOS has received Orphan Drug Designation (ODD) from the FDA for its investigational therapy BA-101 aimed at treating Glioblastoma (GBM) [1][3] - GBM is characterized as an aggressive brain tumor with a median survival of less than 12 months and limited treatment options [2][4] - The company emphasizes the potential of nitric oxide (NO) modulation in improving treatment outcomes for GBM patients [3][4] Company Overview - NeuroNOS focuses on developing therapies for neurological disorders and neuro-oncology, utilizing small molecules to regulate NO levels in the brain [5] - The company has a commitment to advancing medical innovation through collaborations with research institutions and experts [5] - Beyond Air, the parent company of NeuroNOS, is involved in harnessing NO for treating various health conditions, including respiratory illnesses and neurological disorders [6][7] Industry Context - Glioblastoma represents a significant unmet medical need, with current treatments failing to provide curative options [2][4] - Research indicates that dysregulated NO signaling plays a role in GBM biology, suggesting that targeting NO could enhance treatment efficacy [4] - The FDA's ODD provides incentives for developing treatments for rare diseases, including market exclusivity for seven years upon approval [3]