A New Chapter For Autism Care: NeuroNOS Wins FDA Backing For Rare Genetic Disorder Drug

NeuroNOS, a subsidiary of Beyond Air, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-102, for the treatment of Phelan-McDermid Syndrome (PMS). PMS is a rare genetic neurodevelopmental disorder commonly associated with Autism Spectrum Disorder (ASD). The company plans to initiate first-in-human clinical trials for BA-102 in the United States in 2026.

PMS is most often caused by mutations or deletions in the SHANK3 gene and is characterized by symptoms including developmental delays, intellectual disability, limited speech, and autism-related features. There are currently no FDA-approved therapies specifically for PMS, representing a significant unmet medical need in the rare disease space.

Amir Avniel, CEO of NeuroNOS, said in a statement, “Receiving orphan drug designation from the FDA is a significant step forward for this autism program, as well as our broader mission to bring targeted therapies to individuals and families affected by rare neurodevelopmental conditions. By focusing on the genetic underpinnings of Phelan-McDermid Syndrome-a syndromic form of autism-we aim to address the root cause of symptoms and offer new hope where few options currently exist.”

Prof. Haitham Amal, CSO of NeuroNOS, stated, “Phelan-McDermid Syndrome represents a critical unmet medical need. We are committed to working closely with the FDA, patient advocacy groups, scientific foundations, and clinical investigators to accelerate development of a therapy that could meaningfully improve quality of life for those living with this challenging genetic condition.”

The Orphan Drug Designation provides several development incentives, including seven years of market exclusivity upon approval, tax credits for clinical trial costs, waived FDA application fees, and access to regulatory support. NeuroNOS aims to advance BA-102 with support from the FDA and collaboration with patient advocacy organizations and clinical experts.