Aeglea BioTherapeutics’ (NASDAQ:AGLE) ACN00177 received FDA rare pediatric disease designation for the treatment homocystinuria.
Homocystinuria is a serious metabolic disorder characterized by elevated plasma homocysteine, which leads to life-altering complications and reduced life expectancy.
ACN00177 is an engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma. The company initiated a Phase 1/2 clinical trial in the second quarter of 2020 to evaluate ACN00177 for the treatment of homocystinuria.
“Homocystinuria is a serious and progressive disease that significantly impacts the quality of life of patients – with a high risk of life-threatening blood clots, severe vision issues, skeletal abnormalities, developmental delay and intellectual disability,” Anthony Quinn, Ph.D., Aeglea’s president and CEO, said in a statement.
“There is a significant need for new and better therapies for homocystinuria patients given the lack of effective treatment options for many patients. The rare pediatric disease designation speaks to the significant impact of this disease beginning in childhood and is an important incentive in promoting drug development to improve outcomes in this patient population,” he added.
