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TheStreet Open House

LAL Deficiency Disease Review Published In Atherosclerosis

Synageva BioPharma Corp. (Synageva) (NASDAQ:GEVA), a biopharmaceutical company developing therapeutic products for rare diseases, today announced the publication of an overview of lysosomal acid lipase deficiency (LAL Deficiency) in the online version and an upcoming print edition of Atherosclerosis, the official journal of the European Atherosclerosis Society. The publication, entitled “Lysosomal Acid Lipase Deficiency–An Under-Recognized Cause of Dyslipidaemia and Liver Dysfunction,” can be accessed at http://dx.doi.org/10.1016/j.atherosclerosis.2014.04.003.

“LAL Deficiency is an under-recognized disease, where affected individuals may receive no diagnosis or incorrect diagnoses of heterozygous familial hypercholesterolemia, familial combined hyperlipidemia, non-alcoholic steatohepatitis, non-alcoholic fatty liver disease, or cryptogenic cirrhosis,” said Zeljko Reiner, M.D., Director, University Hospital Center, School of Medicine, University of Zagreb, Zagreb, Croatia, and lead author of the study. “Since dyslipidemia is a common clinical manifestation of LAL Deficiency that is shared among other cardiovascular, liver and metabolic diseases and has been associated with accelerated development of atherosclerosis, cardiovascular disease and premature mortality, it is important that physicians remain vigilant to avoid mis- or under-diagnosing this disease. This review paper provides recommendations to lipidologists, endocrinologists, cardiologists, and hepatologists on how to recognize and definitively diagnose individuals with LAL Deficiency using a dried-blood spot test.”

Sebelipase alfa for LAL Deficiency

LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity. LAL Deficiency presenting in children and adults, historically called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis. These complications are due to the buildup of fatty material in the liver, blood vessel walls and other tissues as a result of the decreased LAL enzyme activity. Infants presenting with LAL Deficiency, historically called Wolman disease, show very rapid progression, with death usually in the first six months of life. Affected infants develop severe liver complications, malabsorption, and growth failure.

Sebelipase alfa is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Synageva is evaluating sebelipase alfa in global Phase 3 clinical trials in infants, children and adults with LAL Deficiency. Sebelipase alfa has been granted orphan designation by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (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.

SBC-103 for MPS IIIB

The mucopolysaccharidoses (MPS) consist of a group of rare LSDs caused by a deficiency of enzymes needed to break down complex sugars called glycosaminoglycans. The MPS III syndromes (also known as Sanfilippo syndromes) share complications with other MPS diseases but represent a clinically distinct subset with marked central nervous system degeneration. MPS IIIB, also known as Sanfilippo B syndrome, is caused by a decrease in alpha-N-acetyl-glucosaminidase (NAGLU) enzyme activity which leads to the buildup of abnormal amounts of heparan sulfates (HS) in the brain and other organs. The accumulation of abnormal HS, particularly in the central nervous system, leads to severe cognitive decline, behavioral problems, speech loss, increasing loss of mobility, and premature death.

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