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Synageva is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with life-threatening rare diseases and high unmet medical need. Synageva is currently evaluating sebelipase alfa in global clinical trials for LAL Deficiency, a lysosomal storage disease (LSD) associated with liver damage and accelerated atherosclerosis in children and adults, and in infants, premature death often before six months of age. Sebelipase alfa has been granted orphan designations by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA and was granted Breakthrough Therapy designation by the FDA for infants presenting with LAL Deficiency, historically called Wolman disease.
Synageva’s next program, SBC-103, is an enzyme replacement therapy for another LSD, mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome) for which both the FDA and EMA granted orphan designation. MPS IIIB patients suffer from severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.
In addition to sebelipase alfa and SBC-103, Synageva has multiple other protein therapeutic programs targeting rare diseases in various stages of preclinical development.
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Jan 12, 2015
33rd Annual J.P. Morgan Healthcare Conference
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Jan 12, 2015
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