October 26, 2020 -- Aeglea BioTherapeutics has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for its enzyme therapeutic, ACN00177, for the treatment of homocystinuria.
ACN00177 is a novel recombinant human enzyme therapy designed to lower the total level of homocysteine in blood plasma.
In addition to the FDA's action, the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has issued a positive opinion recommending it for orphan drug designation in the European Union.
Homocysteine accumulation in homocystinuria leads to multiple progressive and serious disease-related complications including thromboembolic vascular events, skeletal abnormalities such as severe osteoporosis, developmental delay, intellectual disability, lens dislocation, and severe near-sightedness.
ACN00177 is designed to degrade the amino acid homocysteine and its related homocysteine dimer. Preclinical data demonstrated that the candidate improved important disease-related abnormalities and survival in a mouse model of homocystinuria.
Aeglea initiated an ongoing phase I/II trial in the second quarter of 2020.