Lomitapide shows positive results in treating homozygous familial hypercholesterolemia


A phase 3 trial of lomitapide showed safety and efficacy for treating homozygous familial hypercholesterolemia (HoFH) in pediatric patients aged 5 to 17 years.

Lomitapide (Juxtapid; Amryt) has shown positive results in the APH-19 phase 3 trial for treating pediatric patients aged 5 to 17 years with homozygous familial hypercholesterolemia (HoFH).

The single-arm, open-label, international, multi-center study analyzed the safety and efficacy of lomitapide in pediatric patients receiving stable lipid-lowering therapy for HoFH. Lomitapide was added to patients’ liquid-lowering therapy established in the study’s run-in phase.

Day 0 of the study’s efficacy phase was when lomitapide was initialized as add-on therapy. Dosage started as the recommended starting dose, eventually increasing to the greatest dose applicable for patients’ age groups.

There were 46 pediatric patients enrolled in the study, with 43 completing the run-in phase and entering the efficacy phase. There were 20 patients aged from 5 to 10 years and 23 patients aged from 11 to 17 years. Of these, genetic confirmation of HoFH was found in 88%. At baseline, the mean low density lipoprotein cholesterol (LDL-C) was 436 mg/dl.

The primary endpoint of the study was a significant average reduction of LDL-C at week 24. Both age groups met this endpoint, with patients aged 5 to 10 years seeing a mean reduction of 57% and patients aged 11 to 17 seeing a mean reduction of 51%. This led to an average overall reduction of 54%.

The secondary endpoint of the study was a noteworthy reduction in key lipid parameters, such as non-high density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), and very low-density lipoprotein cholesterol (VLDL-C). Non-HDL-C saw a percent change at week 24 of 54%, TC a percent change of 50%, and VLDL-C a percent change of 50%. 

Safety profiles were consistent with prior data. There were 41 patients who completed the 24-week efficacy phase, as 2 patients withdrew from the study because of adverse events. Of the 5 patients who reported severe adverse events, only 1 was associated with lomitapide.


Amryt announces positive top line results from phase 3 pediatric trial of lomitapide in HoFH. Amryt Pharma. January 5, 2023. Accessed January 9, 2023. https://otp.tools.investis.com/clients/uk/amryt_pharmaceuticals_dac1/usn/usnews-story.aspx?cid=1375&newsid=87034

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