Kanuma (sebelipase alfa) is filed at FDA for LAL deficiency- Synageva Biopharma

Synageva Biopharma recently announced that the FDA accepted for filing the Biologics License Application (BLA) for Kanuma (sebelipase alfa) for the treatment of lysosomal acid lipase deficiency (LAL deficiency). The FDA granted the company's request for Priority Review, which shortens the regulatory review period and is reserved for investigational therapies that treat serious conditions and, if approved, would provide a significant improvement in safety or effectiveness compared to available therapies.

The FDA established a target action date of September 8, 2015 under the Prescription Drug User Fee Act (PDUFA). The FDA indicated that they are not currently planning to hold an advisory committee meeting for the Kanuma BLA. Synageva plans to expand its regulatory submissions for Kanuma beyond the BLA in the U.S. and the marketing authorization application in Europe to include Mexico and Japan during 2015.

Comment: Kanuma was filed at EMA for LAL deficiency in December 2014. LAL deficiency (LAL Deficiency )is a serious, underdiagnosed disease that manifests with significant morbidity and early mortality and can lead to sudden and unpredictable clinical complications. LAL deficiency often manifests in childhood, but can be diagnosed at all ages with a simple blood test. 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.