Athersys (ATHX) MultiStem Granted Orphan Drug Designation by FDA for Hurler's Syndrome Treatment
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Athersys, Inc. (Nasdaq: ATHX) announced that MultiStem®, Athersys' proprietary cell therapy, has been granted orphan drug designation by the U.S. Food and Drug Administration for the treatment of Hurler's Syndrome, also known as mucopolysaccharidosis type I or MPS-I. Hurler's Syndrome is a lysosomal storage disorder that affects approximately 1 of every 100,000 children. Orphan drug designation, which is intended to facilitate drug development, 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.
Lysosomal storage diseases are a group of rare, inherited diseases that initially result from defects in the lysosome, a component of the cell that is responsible for the breakdown of certain molecules, such as complex lipids and sugars. In such cases, the lysosome produces too little of an enzyme that is necessary for the break-down of these materials, resulting in unwanted accumulations in the cells and tissues, which can lead to neurological disease and a range other complications. Hurler's Syndrome 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.
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Lysosomal storage diseases are a group of rare, inherited diseases that initially result from defects in the lysosome, a component of the cell that is responsible for the breakdown of certain molecules, such as complex lipids and sugars. In such cases, the lysosome produces too little of an enzyme that is necessary for the break-down of these materials, resulting in unwanted accumulations in the cells and tissues, which can lead to neurological disease and a range other complications. Hurler's Syndrome 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.
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