Synageva BioPharma's Sebelipase Alfa Receives Breakthrough Therapy Designation for Early Onset LAL Deficiency From the FDA

Published: May 20, 2013

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LEXINGTON, Mass.--(BUSINESS WIRE)--Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to sebelipase alfa for the treatment of early onset lysosomal acid lipase deficiency (LAL Deficiency), also known as Wolman disease. The designation was based on data generated from clinical trials with sebelipase alfa in patients with early onset LAL Deficiency. The FDA also confirmed that late onset LAL Deficiency is “a serious and life threatening disease or condition” and that Breakthrough Therapy designation could be obtained for this aspect of the disease with additional clinical information.

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