Athersys has received FDA orphan drug designation for its proprietary cell therapy, MultiStem, to treat Hurler's syndrome, also known as mucopolysaccharidosis type I or MPS-I.
The orphan drug designation provides substantial potential benefits to the sponsor, including funding for certain clinical studies, study-design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.
The preclinical results of the study conducted by the University of Minnesota investigators together with Athersys scientists suggest that MultiStem cells could provide benefit to patients suffering from lysosomal storage disorders, such as Hurler's Syndrome.
The study demonstrated that an injection of MultiStem cells in neonatal MPS-I mice reduces the accumulation of GAGs in the brain thereby resulting in improvements in behavioral and motor function in the diseased mice.
Hurler's Syndrome, a lysosomal storage disorder, is caused by a deficiency of a specific enzyme responsible for degrading complex sugars called glycosaminoglycans (GAGs), accumulation of which can lead to mental retardation, respiratory and cardiac complications, and death in early childhood.
