Interim Data of Kanuma in LAL-D Infants Presented at WORLDSymposium

This morning at the 14^th Annual WORLDSymposium in San Diego, new combined interim data from two ongoing open-label studies were presented, exhibiting a 3-year survival estimate of 68% in infants with rapidly progressing lysosomal acid lipase deficiency (LAL-D) treated with Kanuma (sebelipase alfa).

Alexion Pharmaceuticals, Inc. announced that the data from two ongoing open-label studies — VITAL and CL-08 – clearly prove the significant clinical benefit of the drug in this early-onset patient population.

In patients with this autosomal recessive disease, a mutation in the LIPA gene results in the lack of the LAL enzyme. Without it — or with insufficient amounts of it – fat is unable to be processed inside the lysosomes, leading to a buildup within the cells and tissues of the body. Symptoms typically vary from patient to patient, based on how much of the enzyme is present.

If the enzyme is completely nonexistent, symptoms, including severe liver and adrenal gland damage, begin in the first year of life.

“Rapidly progressive LAL-D previously meant a death sentence for most infants diagnosed with this devastating and ultra-rare metabolic disease,” said John Orloff, M.D., Executive Vice President and Global Head of R&D at Alexion. “It is gratifying and humbling to see that many of these infants are surviving into childhood when treated with Kanuma, while experiencing important improvements in their disease symptoms.”

Sebelipase alfa, which was approved for use in this indication by the U.S. Food and Drug Administration (FDA) in 2015, is an enzyme replacement therapy that was developed to replace the missing critical enzyme and reduce lipid substrate accumulation in the lysosomes of cells throughout the body.

The VITAL study enrolled 9 patients initiated treatment with sebelipase alfa at a median of 3.0 months of age and received a starting dose of 0.35 mg/kg once-weekly for the first 2 weeks, with dose escalation up to 1, 3 or 5 mg/kg, per protocol. Patients in the CL08 study (10 patients) initiated treatment with the enzyme replacement therapy at a median of 2.8 months of age and received 1 mg/kg once-weekly with dose escalation up to 3 or 5 mg/kg, per protocol.

Between the two studies, a total of 19 infants were enrolled, 7 of whom are surviving and have reached 3 years of age. An additional 6 infants who have not yet reached that age have also shown clean benefit from improvements in key parameters, including weight gain and markers of liver disease and function. None of the enrolled participants discontinued due to adverse events (AEs).

A total of 6 patient deaths were reported between the two studies, none of which were considered by investigators to be related to Kanuma.

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