Benzinga | Aug 3, 2020 07:03AM EDT

Cerecor Receives Orphan Drug Designation For CERC-006 In Lymphatic Malformations

ROCKVILLE, Md. and CHESTERBROOK, Pa., Aug. 03, 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 Orphan Drug Designation (ODD) to CERC-006, a dual inhibitor of mTOR complexes 1 and 2 for the treatment of lymphatic malformations (LM).



Lymphatic malformations are a family of rare, congenital neoplastic diseases caused by mutations in certain genes that regulate cell growth in the lymphatic system. Children who suffer from this condition often have life-altering and in severe cases, life-threatening accumulation of fluid or growth affecting the head, neck and vital organs. LM appears at birth or in infancy and may persist throughout life. It is estimated that 30,000 to 60,000 Americans have this condition.1Currently, there are no approved medicines for LM and affected children must often endure painful and potentially disfiguring surgery or sclerosis. As many as 20% of afflicted children die within 7 years.2