Sebelipase alfa (SBC-102) is a recombinant form of the human lysosomal acid lipase (LAL) enzyme under development by Synageva as an enzyme replacement therapy for LAL Deficiency. LAL Deficiency is a rare autosomal recessive lysosomal storage disease (LSD) caused by a marked decrease in LAL enzyme activity that results in the buildup of fatty material in the liver, blood vessel walls, and other tissues and organs. This buildup of fatty material often results in significant morbidity and early mortality in infants, children, and adults. LAL Deficiency in children and adults, sometimes called Cholesteryl Ester Storage Disease (CESD), is an underappreciated cause of cirrhosis and accelerated atherosclerosis. Infants with LAL Deficiency, sometimes called Wolman disease, suffer from the most rapidly progressive form of LAL Deficiency, which is usually fatal within the first six months of life due to severe malabsorption, growth failure, and liver failure. There are no approved therapies 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 designations 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 infants with LAL Deficiency.
For more information about our clinical trial programs for sebelipase alfa, please visit clinicaltrials.gov (search term: sebelipase).
For more information about LAL Deficiency, including common signs and symptoms, diagnostic information and testing resources, clinical trial options, and options for support for patients and caregivers please visit www.LALDeficiencySource.com.