FDA grants orphan drug designation for Synageva BioPharma's SBC-102

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the U.S. 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 FDA filing and registration fees, and seven years of market exclusivity upon approval. U.S. 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.


The opinions expressed here are the views of the writer and do not necessarily reflect the views and opinions of News Medical.
Post a new comment
You might also like...
Could a genetic biomarker predict your risk for severe food allergies?