Synageva BioPharma announced that the FDA has granted Breakthrough Therapy designation to sebelipase alfa (SBC-102) for the treatment of early onset lysosomal acid lipase deficiency (LAL Deficiency), also known as Wolman disease. Sebelipase alfa is a recombinant form of the human LAL enzyme under development as an enzyme replacement therapy for LAL deficiency. The submission was based on data from clinical trials of patients with early onset LAL deficiency on sebelipase alfa.
Synageva is currently evaluating sebelipase alfa in global clinical trials for both early and late onset LAL deficiency. Previously sebelipase alfa was granted Fast Track designation by the FDA.
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