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news.Synageva BioPharma has started rolling submission of a biologics license application (BLA) to the US Food and Drug Administration (FDA) for sebelipase alfa to treat patients with lysosomal acid lipase deficiency (LAL Deficiency).
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LAL Deficiency is a serious, under-diagnosed disease with significant morbidity and early mortality.
A rolling submission enables completed portions of the application to be submitted and reviewed on an ongoing basis by the FDA.
The company intends to submit the marketing authorization application (MAA) to the European Medicines Agency (EMA) by the end of January 2015.
Both the BLA and MAA are based on data from the global, randomized, double-blind, placebo controlled Phase III trial of sebelipase alfa in children and adults with LAL Deficiency, and the Phase II/III trial of the drug in infants with LAL Deficiency.
Currently, the company is developing sebelipase alfa, a recombinant form of the human LAL enzyme, as an enzyme replacement therapy for LAL Deficiency.
The drug has secured orphan designation by the FDA, the EMA and the Japanese Ministry of Health, Labour and Welfare.
In addition, the drug received FDA fast track status and breakthrough therapy designation for LAL Deficiency presenting in infants.