AMO Pharma Receives FDA Orphan Drug Designation for AMO-04 in Treatment of Rett Syndrome

Press release from is excited to share that a third compound that was successfully screened in our Scout Program is moving forward into human clinical trials

AMO Pharma have announced that they have received Orphan Drug Designation from the U.S. Food and Drug Administration for their investigational drug candidate AMO-04 for the treatment of Rett syndrome. AMO Pharma is planning the first clinical trial of AMO-04 in individuals with Rett syndrome.

AMO-04 has shown early-stage promise as a treatment for Rett syndrome. In preclinical trials it was well-tolerated, and improved motor coordination and other neurologic and behavioral parameters measured.’s Chief Science Officer, Steve Kaminsky, PhD, comments,

“This is exciting because with each new drug that enters into clinical trials in Rett syndrome, we improve our ability to enhance the quality of life of those living with this disorder.” is excited about AMO-04 and the other successful candidate drugs that have been screened in our Scout Program. Their successes demonstrate the importance of the Scout Program in aggressively accelerating the pace of finding potential treatments for Rett syndrome while we continue our search for a cure.

To read AMO Pharma’s full press release, visit

If you have other questions about this announcement, please refer to our Q&A document at

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