Cerecor Receives Rare Pediatric Disease Designation for CERC-006 in Lymphatic Malformations

ROCKVILLE, Md. and CHESTERBROOK, Pa., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Cerecor Inc. (NASDAQ: CERC), a leading biopharmaceutical company in the development and commercialization of treatments for rare pediatric and orphan diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Rare Pediatric Disease Designation (RPDD) to CERC-006, a dual inhibitor of mTOR complexes 1 and 2 for the treatment of lymphatic malformations (LM).

Lymphatic malformations are rare, non-malignant masses consisting of fluid-filled channels or spaces thought to be caused by the abnormal development of the lymphatic system. LM occurs mostly in infancy or early childhood and can persist throughout life. It is estimated that 30,000 to 60,000 Americans have this condition in the United States.¹

“We are very pleased to receive FDA Rare Pediatric Disease Designation for CERC-006. This designation underscores the high unmet need in this family of serious and rare diseases of infants and children that so often lead to disability and in some cases, death,” said Mike Cola, Chief Executive Officer, Cerecor

The FDA grants RPDD to programs addressing rare diseases or conditions that are serious or life-threatening in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years.  The term “rare disease or condition” means any disease or conditions affecting less than 200,000 people in the United States. If a new drug application (NDA) for CERC-006 is approved, Cerecor would be eligible to receive a priority review voucher for another compound, which it could use itself, or sell to another company.

About CERC-006

CERC-006 is an orally available blocker of mTOR complex 1 and 2 that is being developed for the treatment of serious lymphatic malformations (LM) not treatable with surgery or sclerotherapy. Because a large majority of LM patients have activating mutations in the PK/AKT/mTOR pathway, we believe CERC-006 has the potential to specifically reduce proliferation of the abnormal cells that cause LM, reduce the size of neoplastic lesions and restore lymphatic function; ultimately improving and prolonging the lives of many affected children. A Phase 1b/2a proof-of-concept trial to test the safety and efficacy of CERC-006 in LM is planned to begin in 2021.