Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, announced today the submission to the Comisión Federal para la Protección contra Riesgos Sanitarios (Cofepris) in Mexico for Kanuma as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency), a rare genetic disease with significant morbidity and early mortality. The submission to Cofepris for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency.
Kanuma and LAL Deficiency
LAL Deficiency is a serious and life-threatening disease that can be diagnosed with a simple blood test. LAL Deficiency causes progressive and multisystemic organ damage including cirrhosis and accelerated atherosclerosis that can lead to sudden and unpredictable clinical complications. LAL Deficiency often manifests in childhood but can be diagnosed at all ages. LAL Deficiency is caused by genetic mutations that result in decreased LAL enzyme activity in the lysosomes across multiple body tissues, leading to the buildup of fatty material in the liver, blood vessel walls and other tissues.
Kanuma is a recombinant form of the human LAL enzyme being developed by Synageva as an enzyme replacement therapy for LAL Deficiency. Kanuma has been granted orphan designation by the FDA, the European Medicines Agency (EMA), and the Japanese Ministry of Health, Labour and Welfare. Additionally, Kanuma received fast track designation by the FDA, and Breakthrough Therapy designation by the FDA for LAL Deficiency presenting in infants. The FDA accepted for review the BLA for Kanuma and granted the company's request for Priority Review. The EMA validated the Marketing Authorization Application (MAA) for Kanuma and granted the company's request for accelerated assessment.