Breakthrough Therapy for Rare Enzyme Disorder Could Be Approved Even Sooner
Synageva BioPharma announced that the FDA has accepted for Priority Review the Biologics License Application (BLA) for Kanuma (sebelipase alfa), a recombinant form of the human LAL enzyme, for the treatment of lysosomal acid lipase deficiency (LAL Deficiency).
The BLA submission for Kanuma included data from the global, randomized, double-blind, placebo controlled Phase 3 trial in children and adults with LAL Deficiency, and the Phase 2/3 trial of Kanuma in infants with LAL Deficiency. Patients in these trials, combined with patients in other ongoing clinical trials with Kanuma, represent the largest patient population studied to date for this rare disease.
Previously, Kanuma has been granted Orphan Drug designation. Additionally, Kanuma received Fast Track designation by the FDA, and Breakthrough Therapy designation for LAL Deficiency presenting in infants.
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