Alexion Pharmaceuticals has presented new long-term data from an ongoing, open-label extension of the pivotal Phase 3 ARISE trial of Kanuma ® (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease.

At 52 weeks of Kanuma treatment, nearly all patients (97 percent) who had received Kanuma from the start of the double-blind period had a rapid and sustained reduction in alanine aminotransferase.