Nitric Oxide-Regulating Therapy Gets Orphan Drug Status for Syndromic Autism

The Food and Drug Administration (FDA) has granted Orphan Drug designation to BA-102 for the treatment of Phelan-McDermid syndrome, a rare neurodevelopmental disorder linked to autism.

Phelan-McDermid syndrome is caused by a deletion or mutation in the SHANK3 gene on chromosome 22. There are currently no FDA-approved treatments for this genetic disorder, which is characterized by intellectual disability, severely delayed speech, motor impairments, hypotonia, and behaviors associated with autism spectrum disorder (ASD).

BA-102 is an investigational agent that aims to target and regulate nitric oxide (NO) levels in the brain. Based on preclinical studies, it is believed that NO dysregulation contributes to ASD and that balancing NO levels may help improve behavioral and cognitive function in these patients. A first-in-human clinical trial is expected to begin in the US in 2026.

“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,” said Amir Avniel, CEO of NeuroNOS. “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.”

The FDA’s Orphan Drug designation is granted to medicines intended to treat or prevent rare diseases or disorders that affect fewer than 200,000 individuals.

This article originally appeared on MPR