Cerecor Inc. said three of its therapies for metabolism-related disorders received orphan-drug designations from the U.S. Food and Drug Administration.
The FDA granted orphan-drug status to CERC-801, D-galactose for treating phosphoglucomutase 1 deficiency; CERC-802, D-mannose to treat mannose phosphate isomerase deficiency; and CERC-803, L-fucose for the treatment of congenital disorder of glycosylation 2c.
Each indication is an ultra-rare congenital disorder of glycosylation — a disease caused by an inherited mutation — affecting fewer than 1,000 individuals in the U.S., the Baltimore-based biopharmaceutical company said. Glycosylation is a process that creates carbohydrates needed for tissues and organs to function.
Cerecor completed pre-investigational new drug application meetings with the U.S. drug agency and seeks to expedite the approval of each product.