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news.US-based biopharmaceutical firm Synageva BioPharma has submitted a new drug application (NDA) to the Ministry of Health, Labour and Welfare (MHLW) in Japan for Kanuma (sebelipase alfa) to treat patients with lysosomal acid lipase deficiency (LAL Deficiency).
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Kanuma would be the first approved therapy for these patients if it secures approval from the Ministry.
The NDA was based on data from the global, randomized, double-blind, placebo controlled Phase III trial in children and adults with LAL Deficiency, which included enrollment of Japanese patients, and the Phase II/III trial of Kanuma in infants with LAL Deficiency.
Earlier, the MHLW had granted orphan drug designation (ODD) to sebelipase alfa for the treatment of LAL Deficiency as a result, this NDA will receive priority review for marketing authorization.
If approved, sebelipase alfa would have ten years of market exclusivity for the designated indication.
Kanuma is a recombinant form of the human LAL enzyme currently being developed by the company as an enzyme replacement therapy for patients with LAL Deficiency.
The drug has also secured orphan drug status from the US food and Drug Administration (FDA), the European Medicines Agency (EMA) and the Japanese MHLW.
In addition, Kanuma secured fast track designation and breakthrough therapy designation from the FDA for LAL Deficiency presenting in infants.