On August 2, 2018, ArmaGen, Inc., a biopharmaceutical company located in Calabasas, California, announced that the U.S. Food and Drug Administration’s Office of Orphan Products Development has granted Orphan Drug Designation to its investigational agent AGT-184 for the treatment of lysosomal disease mucopolysaccharidosis type IIIA (also known as MPS IIIA, or Sanfilippo syndrome type A). AGT-184 is an investigational enzyme replacement therapy for the treatment of the cognitive effects of MPS IIIA. The missing or diminished enzyme needing therapeutic replacement is N-sulfoglucosamine sulfohydrolase (“SGSH”).
AGT-184 is an immunoglobulin G (“IgG”)-SGSH fusion-protein, where the IgG portion is a human anti-insulin receptor monoclonal antibody. The insulin receptor antibody domain triggers transport of the AGT-184 fusion-protein across the blood brain barrier, by binding to endogenous insulin receptors present on the blood brain barrier. Normally, the blood brain barrier blocks large molecules such as enzymes from entering the brain. In effect, this is a Trojan Horse approach to successfully delivering the therapeutic agent to the brain. ArmaGen, Inc. is currently conducting Investigational New Drug (“IND”) application-enabling activities (manufacturing, toxicology studies), with the goal of filing an IND application with the U.S. Food and Drug Administration in late 2019.
The FDA grants Orphan Drug Designation to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. This designation confers special financial incentives to the drug developer, including tax credits on clinical development costs and prescription drug user-fee waivers, and it may confer the right to seven years of market exclusivity in the U.S. upon FDA approval of the orphan drug.