AMO Pharma announced the U.S. Food and Drug Administration has granted Orphan Drug Designation to its investigational therapy AMO-04 for the treatment of Rett syndrome, a rare childhood neurodevelopmental disorder.
"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. "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."
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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 (MECP2) gene. It is often misdiagnosed as autism, cerebral palsy, or non-specific developmental delay because patients typically experience problems with cognitive, sensory, emotional, motor and autonomic function. Other symptoms include seizures, disorganized breathing patterns while awake, scoliosis and sleep disturbances.
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 that is sponsored by Rettsyndrome.org. Additional research conducted by Numedicus, a private biopharmaceutical company focused on the repurposing of drugs, also indicated AMO-04 could benefit patients living with certain breathing disorders. AMO Pharma has an exclusive license agreement with Numedicus covering AMO-04 and related compounds.