Positive top line results from phase III pediatric trial of lomitapide in HoFH.- Amryt.

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 <0.0001 (95% cl: -62% to -45%, p><0.0001) from baseline to week 24 in ldl-c, for the overall group of patients (n="43" patients).>This was consistent between the two pre-specified age groups : i. Patients aged 5 to 10 years (n=20): a mean reduction of 57% (95% CI: -68% to -45%) from baseline to week 24 in LDL-C ii. 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. Key Secondary Endpoints : Statistically meaningful reduction in key lipid parameters: Percent change at week 24 compared to baseline in the following lipid parameters: i. Mean reduction in non-high density lipoprotein cholesterol (non-HDL-C) : 54% (95% CI -62, -45%), p <0.0001. ii. mean reduction in total cholesterol (tc) : 50% (95% ci -58, -42%), p><0.0001. iii. mean reduction in very low-density lipoprotein cholesterol (vldl-c) : 50% (95% ci -59, -41%), p><0.0001.></0.0001.></0.0001.></0.0001.></0.0001)></0.0001>

Safety was consistent with the known profile of lomitapide, no new signal identifiedi. 41 patients completed the 24-week efficacy phase. 2 patients withdrew due to adverse events ii. 5 patients had serious adverse events, with only 1 considered to be related to lomitapide

About APH-19 Trial :The Phase III study was a single-arm, open-label, international, multi-center study to evaluate the efficacy and safety of lomitapide in pediatric patients with Homozygous Familial Hypercholesterolemia (HoFH) on stable lipid-lowering therapy. Approximately 45 pediatric patients with HoFH were to be treated with lomitapide, added to their current stable lipid lowering therapy (LLT) (including lipoprotein apheresis (LA), when applicable) that was established during the Run-in Period. After the stabilization of the patient at their Maximum Tolerated Dose (MTD) of LLT (including LA, when applicable) during the 6-week Run-in Period, treatment with lomitapide was started as an add-on therapy on Day zero of the Efficacy Phase. Dosing of lomitapide was initiated at the recommended starting dose and escalated to the maximum dose applicable to their age group based upon safety and tolerability in addition to LDL-C values. During the 24-week Efficacy Phase, patients were required to remain on their stable LLT regimen (including LA, when applicable) established during the 6-week Run-in Period. Enrolment was stratified to ensure an adequate number of patients were enrolled in the following age groups: 5 to 10 years and 11 to less than 17 years.