18 June 2018 - - Berwyn, Pennsylvania- and London, England-based biopharmaceutical company AMO Pharma has been notified that the US Food and Drug Administration (FDA) has granted orphan drug designation to its investigational therapy AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder, the company said.
Rett syndrome is a neurological disorder that is typically first recognized in infancy and seen almost always in girls.
It is caused in most cases by mutations in the X-linked methyl-CpG-binding protein 2 gene. Patients typically experience problems with cognitive, sensory, emotional, motor, and autonomic function.
AMO-04 is a glutamate modulator that has shown early-stage promise in the treatment of Rett syndrome based on screening by the Scout Program, a drug discovery screen in a mouse model of the disease.
Privately held AMO Pharma is working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare, debilitating childhood onset neurogenetic disorders with limited or no treatment options.