Synageva BioPharma is a biopharmaceutical company dedicated to discovering, developing and delivering medicines for patients with rare disease and high unmet medical need. At Synageva, we share a passion and commitment to improving the lives of patients with rare diseases. Our team is drawn from leading biotech companies and has helped bring several life-saving therapies to patients whose diseases were once considered too rare for developing treatments. We believe that every patient deserves treatment options, regardless of the rarity of their disease.
Synageva's sebelipase alfa (SBC-102) is a recombinant form of the human lysosomal acid lipase (LAL) enzyme under development 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. Reduced LAL enzyme activity results in the accumulation of fatty material in the liver, blood vessel walls and other tissues and organs that can often lead to the clinical manifestations associated with the disease.
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 that is usually fatal within the first six months of life. Affected infants develop 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. 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 early onset LAL Deficiency.
Synageva's SBC-103 is a recombinant form of the human alpha-N-acetyl-glucosaminidase (NAGLU) enzyme under development as an enzyme replacement therapy for mucopolysaccharidosis IIIB (MPS IIIB, also known as Sanfilippo B syndrome). MPS IIIB is caused by a marked decrease in 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. Using various dosing approaches, SBC-103 reduced HSD substrate storage in the brains, liver and kidney tissues in an MPS IIIB animal model. SBC-103 has been granted orphan designation by the FDA and the EMA.
In addition to sebelipase alfa and SBC-103, Synageva has several other protein therapeutic programs targeting rare diseases in various stages of preclinical development and include programs beyond the lysosomal storage disorders. We selected these programs based on scientific rationale, unmet medical need, potential to impact disease course and strategic alignment with our corporate focus.
Synageva’s focus on developing, delivering and commercializing products for rare diseases was established in 2008 with the appointment of Sanj K. Patel as President and CEO. The formation of Synageva and its change to a product-based company represented a considerable shift in the business strategy of the original company, AviGenics, Inc., which was founded in 1996 and was focused on the development of a novel protein production technology. Today, many of Synageva’s product candidates are produced using this proprietary expression system, which is based on over 15 years of research and clinical development. Synageva’s proprietary technology is highly consistent and scalable, allowing the rapid initiation and simultaneous advancement of multiple programs, enabling Synageva to perform initial investigations of programs in multiple diseases.
Patients and Families: Click here for more information on LAL Deficiency, or contact Synageva here.
Physicians: Click here to contact Synageva if you have a patient with LAL Deficiency or if you are interested in participating in research and clinical trials related to LAL Deficiency.