Sangamo announces European Medicines Agency designation for MPS I and MPS II
Sangamo Therapeutics announced that the Committee for Orphan Medicinal Products of the European Medicines Agency has issued a positive opinion on the application for orphan medicinal product designation for SB-318 and SB-913, Sangamo's genome editing product candidates for the treatment of rare lysosomal storage disorders Mucopolysaccharidosis Type I - MPS I - and MPS II, respectively. The EMA's OMPD is granted to medicines intended for the treatment, prevention or diagnosis of life-threatening or chronically debilitating conditions that are rare and affect less than five in 10,000 persons in the European Union. The designation provides incentives to advance the development and commercialization of orphan medicines, which include access to the EU centralized authorization procedure and potential for market exclusivity for a period of up to ten years. Using Sangamo's zinc finger nuclease genome editing technology, SB-318 and SB-913 are designed as a single treatment strategy intended to provide stable, continuous production of the IDUA or IDS enzyme for the lifetime of the patient.