Rare Disease Report

FDA Grants Orphan Drug Designation to New Treatment for Rett Syndrome

JUNE 15, 2018
Krista Rossi
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.

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