Lomitapide was associated with statistically significant reductions in low-density lipoprotein cholesterol (LDL-C) in children with homozygous familial hypercholesterolemia (HoFH), according to topline results from a phase 3 trial.

The single-arm, open-label APH-19 trial (ClinicalTrials.gov Identifier: NCT04681170) enrolled a total of 46 patients 5 to 17 years of age with HoFH who had a mean LDL-C at baseline of 436mg/dL (SD 189). After the 6-week run-in period, 43 patients received lomitapide, in addition to their current stable lipid lowering therapy (including lipoprotein apheresis, when applicable).

Results showed that treatment with lomitapide met the primary endpoint demonstrating a statistically significant mean reduction of 54% in LDL-C from baseline to week 24 (95% CI, -62%, -45%; P <.0001). For patients 5 to 10 years of age (n=20), the mean reduction in LDL-C from baseline to week 24 was 57% (95% CI, -68%, -45%). For patients 11 to 17 years of age (n=23), the mean reduction in LDL-C from baseline to week 24 was 51% (95% CI, -62%, -40%).

Additionally, statistically meaningful reductions in non-high density lipoprotein cholesterol (54% [95% CI, -62%, -45%]; P <.0001), total cholesterol (50% [95% CI, -58%, -42%]; P <.0001), and very low-density lipoprotein cholesterol (50% [95% CI, -59%, -41%]; P <.0001) were observed.

The safety of lomitapide was found to be consistent with its known profile; no new safety signals were identified in the pediatric trial.

Lomitapide is currently available under the brand name Juxtapid as an adjunct to low-fat diet and other lipid-lowering treatments, including LDL apheresis where available, to reduce LDL-C, total cholesterol, apo B, and non-HDL-C in adults with HoFH.


Amryt announces positive top line results from phase 3 pediatric trial of lomitapide in HoFH. News release. Amryt Pharma plc. Accessed January 5, 2022. https://www.globenewswire.com/news-release/2023/01/05/2583508/0/en/Amryt-Announces-Positive-Top-Line-Results-from-Phase-3-Pediatric-Trial-of-Lomitapide-in-HoFH.html.