Synageva BioPharma is a biopharmaceutical company with headquarters, research and development facilities in Lexington, MA. Synageva was formed to focus on novel orphan treatments for rare diseases. Our lead program, sebelipase alfa, is a recombinant human LAL (rhLAL), an enzyme replacement therapy for LAL Deficiency. Sebelipase alfa is currently in global clinical development and has been granted orphan designations by the US Food and Drug Administration ("FDA"), the European Medicines Agency, 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.  LAL Deficiency is a rare, serious and devastating lysosomal storage disorder (LSD) that results in significant morbidity and mortality. Synageva has four additional orphan products in development. To ensure that these therapeutic candidates reach patients in need, Synageva has recruited a team with a proven record of discovery, development and commercial experience within rare diseases.

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Our work is based on creating value for patients and their healthcare providers, our shareholders and our employees. Our success brings new treatments to patients and providers. We are building a sustainable business and value for our shareholders because good medicine is good business. Our culture attracts and retains principle-minded individuals of integrity, drive, energy and passion.  Synageva encourages our inspired and talented employees to experience the pride that comes from contributing to the development of life changing therapies.

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Watch this video to learn more about Lysosomal Acid Lipase (LAL) Deficiency, both early and late onset forms.  You will hear the perspective of two mothers who have personal experience with the early onset form of LAL Deficiency, which is often referred to as Wolman Disease.  There are currently no approved treatments for this rare and devastating lysosomal storage disorder.  Synageva BioPharma is developing sebelipase alfa (SBC-102) as an enzyme replacement therapy for LAL Deficiency.  For more information regarding Synageva's clinical trials, please contact: clinicaltrials@synageva.com (If you are having trouble viewing this video, click here.)

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