Synageva BioPharma Corp, a privately held biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has granted orphan drug designation for SBC-102, the company’s enzyme replacement therapy in development to treat Lysosomal Acid Lipase (LAL) Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease (CESD), a condition for which there is currently no approved treatment.
As a result of the orphan drug designation, Synageva BioPharma will be eligible to receive a number of benefits, including access to grant funding for clinical trials, tax credits, waiver of the US FDA filing and registration fees, and seven years of market exclusivity upon approval. US orphan drug designation is granted to a product that treats a rare disease, a condition that affects fewer than 200,000 Americans.
Synageva BioPharma Corp is dedicated to discovering, developing and commercializing therapies for patients with rare conditions and high unmet medical need.