Synageva BioPharma Corp., a privately held biopharmaceutical company developing therapeutic products for rare disorders, announced today that their lead program, SBC-102, an enzyme replacement therapy for Lysosomal Acid Lipase (LAL) Deficiency, currently in clinical trials, has been granted fast track designation by the U.S. Food and Drug Administration (FDA).
“The fast track program is designed to facilitate drug development and expedite the review of drugs to treat serious diseases like LAL Deficiency”
- LAL Deficiency is a serious life-threatening condition. The early onset form of the disease is the most rapidly fatal form, usually within the first year of life. Late onset LAL Deficiency has a more variable presentation with some patients going undiagnosed until complications manifest in late adulthood, while others can present with liver dysfunction in early childhood. LAL Deficiency is associated with significant ill health with shortened life expectancy.
- There are no approved or effective therapies for patients suffering from LAL Deficiency. SBC-102 has the potential to be a disease modifying long-term enzyme replacement therapy for patients (infants, children/adolescents and adults) with LAL Deficiency.
"The fast track program is designed to facilitate drug development and expedite the review of drugs to treat serious diseases like LAL Deficiency," said Anthony Quinn, M.D., Ph.D., Synageva's Chief Medical Officer and Head of R&D. "We are pleased that after reviewing the seriousness of LAL Deficiency and the potential of SBC-102, the FDA has granted fast track designation for this program. We look forward to working closely with the FDA as we complete clinical development to facilitate regulatory review and accelerate the delivery of an effective treatment for patients suffering from this devastating disease."
SBC-102 is a recombinant human lysosomal acid lipase. This enzyme is responsible for the breakdown of cholesteryl esters and triglycerides. Late onset LAL Deficiency, sometimes called Cholesteryl Ester Storage Disease (CESD), affects both children and adults. In these patients, the buildup of fatty material in the liver, spleen and blood vessel walls leads to complications resulting in significant morbidity and mortality. Early onset LAL Deficiency, sometimes called Wolman Disease, causes growth failure in infants and almost always results in death in the first year of life. Synageva has received orphan drug designations for SBC-102 in both the US and EU and human dosing in clinical trials is underway.
SOURCE Synageva BioPharma Corp.