Synageva's SBC-102 enzyme replacement therapy for LAL deficiency granted European orphan product designation

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the European Medicines Agency (EMA) granted orphan product designation for SBC-102 (Recombinant Human Lysosomal Acid Lipase), the Company's enzyme replacement therapy in development to treat Lysosomal Acid Lipase (LAL) Deficiency.

“Our ability to help these patients depends on delivering a high quality therapeutic and high quality services. Synageva is proactively developing the infrastructure to support these patients and their healthcare providers.”

As a result of the EMA orphan product designation, Synageva BioPharma will be eligible to receive access to protocol assistance, direct access to centralized marketing authorization and 10-year marketing exclusivity upon approval, fee reductions or exemptions, and other national incentives. The EMA's orphan medicinal product designation is given to therapies that treat rare diseases, defined as conditions that affect no more than five in 10,000 persons in the European Union.

"The EMA's orphan designation is an important milestone for the development of SBC-102 as a treatment for LAL Deficiency," said Eric Grinstead, Senior Vice President at Synageva BioPharma. "Our ability to help these patients depends on delivering a high quality therapeutic and high quality services. Synageva is proactively developing the infrastructure to support these patients and their healthcare providers."

LAL Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease (CESD), is a rare lysosomal storage disease (LSD) that affects individuals from infancy through adulthood. The disease shortens life and causes significant medical complications including liver failure and death. There are currently no approved treatments for LAL Deficiency.

"Based on the strength of the data that we presented at the American Society of Human Genetics meeting, Synageva is advancing a global clinical development program to evaluate SBC-102's safety and efficacy for patients with LAL Deficiency," said Anthony Quinn, M.D., Ph.D., Synageva's Chief Medical Officer and Head of R&D.