Synageva BioPharma Files for Orphan Drug Designation for SBC-102, Being Developed to Be the First Enzyme Replacement Therapy to Treat Lysosomal Acid Lipase Deficiency – a Progressive, Often Fatal Disease

WALTHAM, Mass.--(BUSINESS WIRE)--Synageva BioPharma Corp., a privately held biopharmaceutical company, today announced its filing with the U.S. Food and Drug Administration (FDA) requesting orphan drug designation for its program, SBC-102. This program is an enzyme replacement therapy in preclinical development to treat lysosomal acid lipase (LAL) deficiency, also known as Wolman disease or cholesteryl ester storage disease (CESD), a condition for which there is currently no effective treatment.
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