Amryt announced positive results from its APH-19 Phase 3 trial of lomitapide for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) in children aged 5-17 years. Top Line Results: A total of 46 pediatric patients were enrolled, with 43 patients completing the run-in phase and entering the efficacy phase. 20 patients were between 5 and 10 years of age.

23 patients were between 11 and 17 years of age. 88% had genetic confirmation of HoFH. The mean low density lipoprotein cholesterol (LDL-C) at baseline was 436 mg/dl (SD 189).

Primary Endpoint: The study met its primary endpoint with a clinically and statistically meaningful result. Percent change in LDL-C at Week 24 compared to baseline: mean reduction 54%, p· Patients aged 11 to 17 years (n=23): a mean reduction of 51% (95% CI: -62% to -40%) from baseline to week 24 in LDL-C.