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Kanuma shows survival benefit in infants with lysosomal acid lipase deficiency

Infants with lysosomal acid lipase deficiency, a rare metabolic disorder, showed increased survival benefit and improved parameters, such as markers for liver diseases and gastrointestinal symptoms, when treated with Kanuma, according to a press release from the manufacturer.

In a phase 2/3 clinical study, Simon A. Jones, MD, consultant at the Central Manchester University Hospitals NHS Foundation Trust, UK, and colleagues evaluated nine infants with lysosomal acid lipase deficiency (LAL-D) who had evidence of growth failure or rapidly progressive disease and assigned them to a treatment regimen with Kanuma (sebelipase alfa, Alexion Pharmaceuticals, Inc.), an enzyme replacement therapy. Overall, 56% of patients lived beyond 2 years of age and had improvements in various markers of liver disease, anemia, gastrointestinal symptoms, weight gain and hepatosplenomegaly, according to the release.

Simon A. Jones

“It is extremely gratifying that the survival benefit of Kanuma in infants with LAL-D persisted with continued treatment, allowing more than half of patients to survive beyond 2 years without experiencing the devastating consequences of disease progression,” Jones said in the release. “We are very pleased that improvements in survival were accompanied by improvements in multiple disease activity parameters, including weight gain, as well as normal development in most patients.”

The majority of adverse events were mild or moderate (92%). Four treatment-related serious adverse events were reported in one patient: pyrexia, pallor, chills and tachycardia, in association with the same infusion. Four deaths occurred, but none were deemed treatment-related.

In another study, 66 patients (children and adults) with LAL-D were assigned an intravenous infusion of 1 mg per kg of body weight dosage of sebelipase alfa or placebo every other week for 20 weeks. Researchers found that sebelipase alfa reduced hepatic and lipid abnormalities among both populations with LAL-D.

The FDA approved sebelipase alfa for the treatment of adult and pediatric patients diagnosed with LAL-D in December 2015.

Disclosure: Healio.com/Hepatology was unable to confirm relevant financial disclosures of Jones at the time of publication.