- NeuroNOS receives FDA Orphan Drug Designation for BA-102 aimed at treating Phelan-McDermid Syndrome (PMS).
- No FDA-approved treatments currently exist for PMS, a disorder related to Autism Spectrum Disorder (ASD).
- First-in-human clinical trials for the treatment are planned to begin in the United States in 2026.
NeuroNOS, a subsidiary of Beyond Air (XAIR, Financial), has achieved a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) granting Orphan Drug Designation for their lead investigational therapy, BA-102. This therapy targets Phelan-McDermid Syndrome (PMS), a rare genetic disorder associated with Autism Spectrum Disorder (ASD), which currently lacks any FDA-approved treatments.
The FDA's Orphan Drug Designation is a crucial acknowledgment, offering several development incentives for BA-102. These benefits include seven years of market exclusivity upon approval, tax credits for qualified clinical trials, waivers for FDA application fees, and access to FDA protocol assistance. This designation not only underscores the unmet medical need for PMS but also aids NeuroNOS in pursuing a more rapid development and approval pathway for their treatment.
Phelan-McDermid Syndrome is characterized by deficiencies or mutations in the SHANK3 gene, leading to developmental delays, intellectual disabilities, and severe speech impairments. With no treatments currently approved, NeuroNOS's strategy aims to target the genetic basis of this syndrome, potentially offering a focused therapeutic approach beyond broader ASD therapies.
While the orphan drug designation marks an important step forward, the development timeline extends to 2026 when the company plans to initiate first-in-human clinical trials in the United States. This timeline indicates that BA-102 remains in the preclinical stage, with further research and development needed before it can be considered for commercialization.
Beyond Air, through its subsidiary NeuroNOS, continues to explore innovative treatment avenues for neurodevelopmental disorders. Through collaborations with leading institutions and leveraging advanced research, the company aims to offer groundbreaking therapies to improve the lives of those affected by these challenging conditions.
