According to a press release from the biopharmaceutical company Cerecor, Inc., the US Food and Drug Administration (FDA) recently agreed to accept the company’s Investigational New Drug (IND) application for its investigational product CERC-801. This experimental drug is in development as a treatment for phosphoglucomutase 1 (PGM1) deficiency. Cerecor is committed to the development of therapies for rare pediatric and neurological diseases.
About PGM1 Deficiency
PGM1 deficiency, which is also known as PGM1 congenital disorder of glycosylation, is a genetic metabolic disorder in which the glycosylation process of a number of lipids and tissue proteins does not occur properly. PGM1 deficiency and related disorders are often lethal at a young age and have no approved treatments. PGM1 deficiency is caused by mutations of the PGM1 gene. The disorder causes a broad range of severe, systemic symptoms, such as immunodeficiency, ataxia, disorders of coagulation, deficits in nervous system development and psychomotor development, organ dysfunction, hypotonia, and physical deformities. There is a dire need for approved treatment options to become available for these poorly known metabolic disorders. At this juncture, PGM1 deficiency and related congenital disorders of glycosylation are ultimately fatal. To learn more about PGM1 deficiency, click here.
CERC-801 is a purified form of D-galactose. This substance is naturally occurring in fruit and dairy products and plays a role in facilitating protein glycosylation, the process which is dysfunctional in patients with PGM1 deficiency. Glycosylation is the process in which carbs are utilized in the modification of proteins in order to change their function. CERC-801 has been granted Rare Pediatric Disease designation and Orphan Drug designation by the FDA.
The completion of an Investigational New Drug application and its approval by the FDA is necessary before a drug developer can begin conducting clinical trials to test the safety and efficacy of an experimental drug. This testing process for CERC-801 will begin with dosing healthy volunteers in order to test tolerability of a single dose. Cerecor hopes to utilized prior data alongside data from the Phase 1 study in order to accelerate the development of CERC-801.