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Synageva BioPharma Corp's Sebelipase Alfa Receives Breakthrough Therapy Designation for Early Onset LAL Deficiency from U.S. Food and Drug Administration

Monday, 20 May 2013 07:01am EDT 

Synageva BioPharma Corp 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. According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for Breakthrough Therapy designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. 

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