-- First-Ever Product Submission for LAL Deficiency
-- Plans to Complete the BLA and MAA by the End of January 2015
LEXINGTON, Massachusetts, Oct. 21, 2014 /PRNewswire/ -- Synageva BioPharma Corp. (Synageva) (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today the start of a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for sebelipase alfa as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic disease with significant morbidity and early mortality. A rolling submission allows completed portions of the application to be submitted and reviewed by the FDA on an ongoing basis. The company anticipates completing the rolling submission of the BLA to the FDA and submitting the Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) by the end of January 2015. The BLA and MAA will include data from the global, randomized, double-blind, placebo controlled Phase 3 trial of sebelipase alfa in children and adults with LAL Deficiency, and the Phase 2/3 trial of sebelipase alfa in infants with LAL Deficiency.
Sebelipase Alfa for LAL Deficiency
LAL Deficiency is a serious, underdiagnosed disease that manifests with significant morbidity and early mortality. LAL Deficiency causes progressive and multisystemic organ damage including hepatic cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications. LAL Deficiency often manifests in childhood but can be diagnosed at all ages with a simple blood test. LAL Deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.
Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Sebelipase alfa has been granted orphan designation by the FDA, the EMA, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants.
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