FDA awards MC-1 "orphan drug title" for treatment of phosphate sterol (amine) oxidase (PNPO) deficiency

Cardiovascular drug company Mediaure announced today that the U.S. Food and Drug Administration (FDA) has awarded MC-1 the "orphan drug" for the treatment of phosphate sterol (amine) oxidase (PNPO) deficiency.
phosphate sterol (amine) oxidase (PNPO) deficiency is an autosomal recessive genetic disease characterized by severe epileptic encephalopathy in the neonatal period, is ineffective for various anti-epileptic drugs, sterol treatment is ineffective or only partially effective.
can be controlled by phosphate acetaldehyde (PLP) single drug, PLP withdrawal after repeated seizures.
most patients have mutations in their PNPO genes.
Medicure will conduct Phase III clinical trials to assess the effectiveness and safety of MC-1 in treating PNPO deficiency.
addition, the European Medicines Agency (EMA) has awarded MC-1 the title of rare pediatric disease for the treatment of seizures associated with PNPO deficiency.
. Albert D. Friesen, CHIEF Executive Officer and Chairman of the Board of Directors, said, "MC-1 is expected to be the first FDA-approved treatment for patients with PNPO deficiency.
the award of orphan medicine and the title of rare pediatric disease from the FDA is a milestone for MC-1."