KANUMA is an innovative enzyme therapy approved for the treatment of patients with lysosomal acid lipase deficiency (LAL-D).1 KANUMA is approved in the United States, European Union and Japan.
KANUMA is the first approved treatment for patients with LAL-D. It is administered via intravenous infusion.
The safety and efficacy of KANUMA based on data from 75 patients (including infant, pediatric, and adult patients) with LAL-D who were treated with KANUMA in two clinical studies and one supporting open-label extension study.1 In a multicenter, open-label, single-arm clinical study of 9 infant patients with LAL-D (patients who presented with rapidly progressive disease within the first 6 months of life) who were treated with KANUMA, 67% (6 of 9) survived beyond 12 months of age compared with 0% of untreated infants (0 of 21) in a historical cohort, all of whom died by 8 months of age.1
In a multicenter, double-blind, randomized, placebo-controlled clinical study of 66 pediatric and adult patients with LAL-D, aged 4 to 58 years, patients treated with KANUMA had larger reductions from baseline in alanine aminotransferase (ALT) values and liver fat content, as measured by MRI, compared to patients treated with placebo. The significance of these findings as they relate to progression of liver disease in LAL-D has not been established. In addition, treated patients had significant improvements in lipid parameters, including LDL-c, triglycerides, and HDL-c, compared to placebo.1
Sixty-five of 66 patients (98%) who participated in the randomized, placebo-controlled trial entered the open-label period in which all patients received KANUMA. During the open-label extension, patients treated with KANUMA for up to 36 weeks demonstrated improvements in lipid parameters, including LDL-c and HDL-c levels, and ALT. The effect of KANUMA on cardiovascular morbidity and mortality has not been established.1
The most common adverse reactions in patients with rapidly progressive disease presenting within the first 6 months of life (>30%) are diarrhea, vomiting, fever, rhinitis, anemia, cough, nasopharyngitis, and urticaria. The most common adverse reactions in pediatric and adult patients (≥8%) are headache, fever, oropharyngeal pain, nasopharyngitis, asthenia, constipation, and nausea.1