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.