The European Medicines Agency issued a positive opinion recommending orphan medicinal product designation for Sangamo Therapeutics Inc.'s genome editing product candidates to treat rare inherited metabolic disorders.
EMA's Committee for Orphan Medicinal Products issued a positive opinion for SB-318 and SB-913 to treat rare lysosomal storage disorders Mucopolysaccharidosis Type I and Type II, respectively.
The disorders are caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans.
Using Sangamo's zinc finger nuclease genome editing technology, SB-318 and SB-913 are designed as a single treatment strategy to provide stable, continuous production of the enzymes for a patient's lifetime.
Both drugs have received orphan drug, fast track and rare pediatric disease designations from the U.S. Food and Drug Administration.