Synageva BioPharma Corp. announced today that the U.S. Food and Drug Administration has granted the company orphan drug designation for its enzyme replacement therapy in development to treat Lysosomal Acid Lipase Deficiency. As a result, the Waltham company will be eligible for various benefits, including access to grant funding for clinical trials, tax credits, waiver of the FDA fees and seven years of market exclusivity upon approval.
The drug labeled SBC-102 is intended as a treatment for the rare childhood diseases Lysosomal Acid Lipase Deficiency, also known as Wolman Disease, and Cholesteryl Ester Storage Disease (CESD),
Synageva BioPharma focuses on discovering, developing and commercializing therapies for patients with rare conditions and high unmet medical need.
In October, the company pulled in a $12 million investment to close on a total financing of $45 million. The latest funding comes from new life sciences-focused investor New Leaf Venture Partners, as well as returning backers.