Synageva BioPharma is a biopharmaceutical company with headquarters, research and development facilities in Lexington, MA. Synageva was formed to focus on novel orphan treatments for rare diseases. Our lead program, sebelipase alfa, is a recombinant human LAL (rhLAL), an enzyme replacement therapy for LAL Deficiency. Sebelipase alfa is currently in global clinical development and has been granted orphan designations by the US Food and Drug Administration ("FDA"), the European Medicines Agency, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received "fast track" designation by the FDA. LAL Deficiency is a rare, serious and devastating lysosomal storage disorder (LSD) that results in significant morbidity and mortality. Synageva has four additional orphan products in development. To ensure that these therapeutic candidates reach patients in need, Synageva has recruited a team with a proven record of discovery, development and commercial experience within rare diseases.
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