Posted by Michael Wonder on 21 Feb 2019
Cerecor receives fast track designation from FDA for CERC-801 for the treatment of PGM1 deficiency
19 February 2019 - Cerecor announced today that the U.S. FDA has designated fast track designation for CERC-801, an ultra-pure, oral formulation of D-galactose currently in development for the treatment of phosphoglucomutase 1 deficiency, also known as PGM1-CDG.
CERC-801 is an ultra-pure formulation of D-galactose, a naturally occurring monosaccharide found in dairy products and fruit. D-Galactose is consumed by the body to provide substrates for protein glycosylation, the process by which carbohydrates are utilised to modify certain proteins as it relates to protein structure and function.
CERC-801 has been granted orphan drug designation and awarded rare paediatric disease designation by the FDA, granting eligibility for receipt of a priority review voucher upon approval of an new drug application.
