FDA Grants Orphan Drug Designation to New Treatment for Rett Syndrome

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMO Pharma Limited’s investigational therapy AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.

AMO-04 is a glutamate modulator; it has displayed early-stage promise for Rett syndrome treatment based on screening by the Scout Program, which is a drug discovery screen in a mouse model of the disease sponsored by Rettsyndrome.org.

Rett syndrome is a neurological disorder characterized by problems with cognitive, sensory, emotional, motor and autonomic function. Disease recognition typically occurs in infancy, but it is often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Mutations in the X-linked methyl-CpG-binding protein 2 (MECP2) gene are linked to the cause of the disease, which occurs almost always in girls.

"This designation is an important milestone in our development program for AMO-04 and reinforces the critical need for a new treatment option for Rett syndrome, which causes devastating neurological regression in patients," said Michael Snape, chief executive officer of AMO Pharma in a recent statement. "We look forward to advancing this program into the clinic and furthering our commitment to this underserved patient population in which there are currently no approved therapies."

AMO-04 has also been indicated to possibly benefit patients living with certain breathing disorders in additional research conducted by Numedicus. AMO Pharma holds an exclusive license agreement with Numedicus covering AMO-04 and related compounds.