5/21/2013

The FDA granted breakthrough therapy status to Synageva BioPharma's experimental drug sebelipase alfa, or SBC-102, as a treatment for early onset lysosomal acid lipase deficiency, also known as Wolman disease. The drug is currently in clinical trials against both early onset and late onset LAL deficiency. Sebelipase alfa previously won orphan designation and fast track status from the FDA.

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