Posted by Michael Wonder on 03 Dec 2020
Aeglea BioTherapeutics receives FDA rare paediatric disease designation for ACN00177 for the treatment of homocystinuria
1 December 2020 - Company eligible to receive priority review voucher upon FDA approval of ACN00177.
Aeglea BioTherapeutics today announced the U.S. FDA has granted rare paediatric disease designation to ACN00177 for the treatment of homocystinuria, a serious metabolic disorder characterised by elevated plasma homocysteine which leads to a wide range of life-altering complications and reduced life expectancy.
ACN00177 is a novel engineered human enzyme therapy designed to lower the total level of homocysteine in the plasma.
