sepiapterin(Pending FDA Approval) Brand and Other Names: Classes:Enzyme Cofactors Print Dosing & Uses AdultPediatric Phenylketonuria Pending FDA approval for phenylketonuria (PKU) Pharmacology Mechanism of Action Dual mechanism of action to increase activity of phenylalanine hydroxylase (PAH) enzyme and red...
PTC expects to submit the sepiapterin NDA to the FDA no later than the third quarter of 2024. Submissions in 2024 are planned in a number of additional key countries where PTC has existing rare disease commercial infrastructure including Brazil and Japan. About SepiapterinSepiapterin (form...