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Funding drugs for rare diseases
Synageva BioPharma Corp. has won orphan drug status from the European Medicines Agency (EMA) for its enzyme replacement therapy, SBC-102, to treat Lysosomal Acid Lipase Deficiency.
The EMA grants orphan status to potential treatments of rare diseases, affecting fewer than five in 10,000 people in the European Union. Under such designation, the Lexington-based biotech may have access to centralized marketing authorization with 10-year marketing exclusivity post-approval, protocol assistance and fee reductions or exemptions.
Synageva’s SBC-102 is intended to treat Lysosomal Acid Lipase Deficiency, a life-shortening disease, with no currently approved treatments, that can cause liver failure and death. The U.S. Food and Drug Administration granted orphan drug designation to SBC-102 in July.
The company expanded its Lexington headquarters in September, moving from Waltham and adding more lab space for its rare disease drug treatments.
Synageva moved its headquarters to Massachusetts in 2008, following 10 years in Atlanta under a former name, AviGenics.
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