Amryt Pharma announces results from Phase 3 pediatric trial of lomitapide

Amryt announces 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. Dr Joe Wiley, CEO of Amryt Pharma, commented: "We are encouraged by these important additional results regarding the efficacy of lomitapide in treating children with HoFH. This is good news for patients and their families." 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 at baseline was 436 mg/dl. 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% from baseline to week 24 in LDL-C, for the overall group of patients. This was consistent between the two pre-specified age groups: Patients aged 5 to 10 years: a mean reduction of 57% from baseline to week 24 in LDL-C; Patients aged 11 to 17 years: a mean reduction of 51% from baseline to week 24 in LDL-C. Safety was consistent with the known profile of lomitapide, no new signal identified: 41 patients completed the 24-week efficacy phase. 2 patients withdrew due to adverse events; 5 patients had serious adverse events, with only 1 considered to be related to lomitapide