The Food and Drug Administration (FDA) has granted Fast Track designation to CERC-802 (Cerecor Inc.) 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 lead to the inability to utilize certain monosaccharides in the production of glycoproteins. MPI deficiency is caused by mutations in the MPI gene responsible for the synthesis of glycoproteins. MPI-CDG generally results in high infant morbidity and mortality, and currently there is no FDA-approved treatments. 

CERC-802 is an ultra-pure formulation of D-mannose that is utilized as a substrate replacement therapy. CERC-802 works by increasing the availability of the metabolic intermediate, D-mannose, for glycoprotein synthesis. The Company believes that CERC-802 will restore metabolic intermediates and facilitate glycoprotein synthesis, maintenance and function.

The FDA has also granted Orphan Drug and Rare Pediatric Disease designations to CERC-802. In July 2019, the Company announced the enrollment of the first patient in the CDG FIRST (Congenital Disorders of Glycosylation Formative Retrospective Study) trial, a non-interventional, retrospective study that follows general principles of periodic assessment of CDG patients in routine practice. The study will collect natural history and treatment-related data of patients diagnosed with different types of CDGs, including MPI-CDG.

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“We believe that the granting of Fast Track designation for CERC-802 is another crucial step in the development of a potential treatment for this ultra-rare condition,” said Dr Simon Pedder, Executive Chairman of the Board for Cerecor. “We’re currently collecting retrospective data through the CDG FIRST trial to support a New Drug Application for a much-needed therapy.”

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