The U.S. Food and Drug Administration Grants Orphan Drug Designation to ACER-001 for the Potential Treatment of Maple Syrup Urine Disease

In August 2014, Acer Therapeutics Inc., a pharmaceutical company developing therapies for serious rare diseases with significant unmet medical need, was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) to sodium phenylbutyrate (ACER-001) for the treatment of Maple Syrup Urine Disease (MSUD).

Approximately 1,000 patients suffer from MSUD in the U.S. and 3,000 patients are affected worldwide. There is no FDA-approved therapy for MSUD, and the condition is sub-optimally managed via a BCAA-restricted diet alone. Despite this diet, patients still experience poor neurological outcomes and social impairment.


Orphan drug designation, which qualifies Acer for various development incentives under the Orphan Drug Act, is provided to drugs and biologics that are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the U.S.