FDA Grants Cerecor’s Three Substrate Replacement Therapies Orphan Drug Designation

Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of therapies for rare and orphan diseases in pediatrics and neurology, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designations (ODD) to Cerecor's three substrate replacement therapies for Congenital Disorders of Glycosylation (CDGs).

The FDA granted ODD to:  CERC-801, D-galactose for the treatment of Phosphoglucomutase 1 (PGM1) Deficiency; CERC-802, D-mannose for the treatment of Mannose Phosphate Isomerase (MPI) Deficiency; and CERC-803, L-fucose for the treatment of Congenital Disorder of Glycosylation IIc (CDG-IIc).

Each indication is an ultra-rare CDG estimated to have fewer than 1,000 patients in the United States. All three programs have previously been granted Rare Pediatric Disease Designation (RPDD) by the FDA. Cerecor completed pre-IND meetings with the FDA and seeks to leverage the 505(b)(2) pathway to expedite approval of each product.

Peter Greenleaf, Cerecor's CEO, stated, "We are proud and excited to be a leader in CDG drug development.  We've advanced the development of each program on multiple fronts and we look forward to continued progress in our collaborations with researchers, physicians and, most importantly, patients. Our goal is to put these therapies in the hands of patients and their caregivers, and we hope the 505(b)(2) pathway continues to provide flexibility and acceleration of these efforts."

There are numerous benefits associated with receipt of both ODD and RPDD, including:

-7-year marketing exclusivity (upon approval) in the United States;
-Tax credits (up to 25% of clinical development costs);
-Waiver of PDUFA Application Fees (filing fees); and
-Rare Pediatric Disease Priority Review Voucher (upon approval) for each compound that has been granted RPDD