ARMAGEN announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead product AGT-182 for the treatment of mucopolysaccharidosis type II (also known as Hunter syndrome or MPS II.) Hunter syndrome is a rare, genetic lysosomal storage disease caused by a deficient or absent enzyme, iduronate-2-sulfatase. It is a life-threatening disease affecting children as young as 2 years of age.
AGT-182 is a human insulin receptor monoclonal antibody-fused iduronate 2-sulfate designed to cross the blood brain barrier (BBB) through the insulin receptors present on the BBB. ARMAGEN is currently preparing an IND for AGT-182 in order to begin clinical investigation in the first half of 2014.
"We are very pleased to receive FDA orphan drug designation for AGT-182. This designation is an important strategic milestone in the development of our program," said James Callaway, Ph.D., Chief Executive Officer of ARMAGEN. "It represents a transformational moment in the Company's evolution as we prepare for the IND and transition from a research-stage to a clinical-stage organization."
Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. This designation confers special incentives to the drug developer, including tax credits on the clinical development costs, prescription drug user fee waivers and may entitle a period of seven year market exclusivity in the US upon FDA approval.
"Through the development of AGT-182, ARMAGEN hopes to address the significant unmet medical need for patients who suffer CNS impairment as a result of Hunter syndrome," said ARMAGEN founder and Chief Scientific Officer, William Pardridge, M.D. "In addition, the Company will continue applying our BBB-penetrating approach to other compounds internally as well as through external collaborations with pharmaceutical partners."