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Synageva BioPharma Submits Kanuma (Sebelipase Alfa) Application For LAL Deficiency In Japan


Synageva BioPharma Corp. (NASDAQ: GEVA), a biopharmaceutical company developing therapeutic products for rare disorders, today announced the submission of a New Drug Application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for Kanuma™ (sebelipase alfa) as a treatment for patients with lysosomal acid lipase deficiency (LAL Deficiency). Upon approval, Kanuma would be the first approved therapy for these patients.

The NDA for Kanuma included previously reported data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, which included enrollment of Japanese patients, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency. The MHLW previously granted Orphan Drug Designation (ODD) to sebelipase alfa for the treatment of patients with LAL Deficiency. As a result, this NDA will receive priority review for marketing authorization, and, if approved, sebelipase alfa would have 10 years of market exclusivity for the designated indication.

Posted-In: News Press Releases


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