Synageva BioPharma Corp

Synageva BioPharma Corp is incorporated in Delaware in 1993. On November 2, 2011, Trimeris, Inc., a Delaware corporation closed a merger transaction, Reverse Merger with Synageva BioPharma Corp., Pursuant to the Merger Agreement, Private Synageva became a wholly owned subsidiary of Trimeris. As a Reverse Merger, Trimeris changed its name to Synageva BioPharma Corp. The Company is a biopharmaceutical company focused on the discovery, development, and commercialization of therapeutic products for patients with rare diseases. The Company has several proteins therapeutic in its pipeline, including enzyme replacement therapies for lysosomal storage diseases (LSDs) and protein therapeutic programs for other rare diseases that are at the stage of preclinical development. Its program, sebelipase alfa is a recombinant human lysosomal acid lipase deficiency (LAL Deficiency), currently evaluating in global Phase 3 clinical trials in infants, children and adults with LAL Deficiency. LAL Deficiency is rare autosomal recessive LSD characterized by decreased LAL enzyme activity leading to intracellular accumulation of lipids. This disease leads to medical complications with significant morbidity and early mortality. Sebelipase alfa has been granted orphan designation by the FDA, the EMA, and the Japanese Ministry of Health, Labour and Welfare. Additionally, sebelipase alfa has received Fast Track Designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. The Company has not yet received approval to market this product and is not currently commercializing any other products. Sebelipase alfa is a recombinant form of the human LAL enzyme under development as an enzyme replacement therapy for LAL Deficiency. The Company is currently enrolling and dosing Phase 3 Trial of Sebelipase Alfa in Children and Adults with LAL Deficiency. It is also pursuing a development strategy for sebelipase alfa to assess safety and tolerability in a broad population of patients, including infants, children, and adults, and to demonstrate efficacy, safety and tolerability of sebelipase alfa in the treatment of LAL Deficiency. Its potential competitors include large pharmaceutical and biotechnology companies and specialty pharmaceutical companies, academic institutions, government agencies, and research institutions. The preclinical studies and clinical testing, manufacture, labeling, storage, record keeping, advertising, promotion, export, and marketing, among other things, of its product candidates and future products, are subject to extensive regulation by governmental authorities in the U.S. and other countries.