Cerecor Receives Fast Track Designation for Rare Metabolic Disorder Therapy
August 22, 2019
The U.S. Food and Drug Administration has granted Fast Track designation to Cerecor for CERC-802, a replacement therapy currently in development for the treatment of Mannose-Phosphate Isomerase Deficiency, also known as MPI-CDG or CDG-1b.
CDGs are a group of rare, inherited, metabolic disorders caused by glycosylation defects that present as a broad range of clinical symptoms, including coagulopathy, hepatopathy, myopathy, hypoglycemia, protein-losing enteropathy and reduced cell counts. People with the defect are unable to utilize certain monosaccharides in the production of glycoproteins. A deletion or misplacement of a sugar subunit produces a dysfunctional glycoprotein, resulting in a myriad of medical issues. Infants born with CDGs have high morbidity and mortality with no FDA-approved treatments.
One variant of CDGs—MPI-CDG—is due to enzymatic deficiency of the mannose-6 phosphate isomerase (MPI enzyme). To date, there have been less than 50 cases diagnosed worldwide. CERC-802 is an ultra-pure formulation of D-mannose, a naturally occurring monosaccharide designed as a replacement therapy to increase the availability of metabolic intermediates for glycoprotein synthesis.
“We continue to work closely with the FDA to advance this development program forward expeditiously,” said Simon Pedder, executive chairman of the board of Cerecor. “We’re currently collecting retrospective data through the CDG FIRST trial to support a New Drug Application for a much-needed therapy.”
CERC-802 has been granted Orphan Drug designation and Rare Pediatric Disease designation by the FDA, making Cerecor eligible to receive a Priority Review Voucher if it is approved.
Author: Rare Daily Staff
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