Glycerol phenylbutyrate granted orphan drug designation for treatment of STXBP1 developmental and epileptic encephalopathy
Stockholm, October 20, 2023 – Immedica today announces that the European Commission has granted an orphan designation for glycerol phenylbutyrate in STXBP1 developmental and epileptic encephalopathy (STXBP1-DEE), following positive opinion from EMA’s Committee for Orphan Medicinal Products (COMP). Orphan designation is granted to medicines that treat, prevent, or diagnose a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of below 5 in 10,000, and with either no satisfactory method of diagnosis, prevention or treatment or with significant benefit to