Advertise With UsAdvertise on our extensive network of industry websites and newsletters.
Get the PBR newsletterSign up to our free email to get all the latest PBR
news.Synageva BioPharma has received FDA's orphan drug status (ODD) for SBC-102, an enzyme replacement therapy in development to treat lysosomal acid lipase (LAL) deficiency, also known as wolman disease and cholesteryl ester storage disease (CESD).
Subscribe to our email newsletter
SBC-102 is a protein designed to be identical to the enzyme that is missing. It is being evaluated in preclinical studies as a potential enzyme replacement therapy, or ERT, which would replace the missing enzyme and help reduce the build-up of cholesteryl esters and triglycerides throughout the body.
Following orphan drug designation, Synageva BioPharma is expected to have 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.
Synageva BioPharma discovers, develops and commercialises therapies for patients with rare conditions and high unmet medical need.