New Drugs and Therapeutics Update: JUXTAPID® (lomitapide) capsules
Prepared by Daniel Mak, MD
On March 3rd, 2026, the FDA approved and expanded indication for JUXTAPID (lomitapide) capsules for pediatric use in children 2 years of age and older with homozygous familial hypercholesterolemia (HoFH).1
The FDA approval for pediatric use was based on the results of a Phase 3, open-label, multicenter study (APH-19). The study evaluated 43 pediatric participants aged 5 to 17 years with HoFH on stable lipid lowering therapy. The diagnosis of HoFH was defined based on (1) genetic confirmation or (2) untreated LDL-C > 500 mg/dL or treated LDL-C ≥ 300 mg/dL together with either cutaneous or tendon xanthoma before age 10 years old or untreated LDL-C levels consistent with heterozygous familial hypercholesterolemia in both parents.2
During a 24-week treatment period, patients were given JUXTAPID® which was then titrated to each participant’s highest tolerated dose, alongside standard-of-care lipid-lowering therapy and a low-fat diet.
Efficacy: The trial demonstrated a mean 49% reduction in low-density lipoprotein cholesterol (LDL-C) from baseline by Week 24. The treatment also resulted in significant decreases in other lipid parameters (non-HDL-C, total cholesterol, VLDL-C, apolipoprotein B, and triglycerides). A subgroup analysis revealed that the mean decrease in LDL-C at Week 24 was 52% for patients aged 5 to 10 years, and 46% for patients aged 11 to 17 years.2
Adverse effects: Gastrointestinal adverse reactions were reported in 72% of the pediatric patients, with the most common being diarrhea, abdominal pain, and vomiting.2
Important Safety Warnings
JUXTAPID® carries a Boxed Warning regarding the risk of hepatotoxicity, as the drug can cause elevations in liver transaminases and increase hepatic fat (hepatic steatosis). Because of these serious liver risks, JUXTAPID is only available through a restricted distribution program called the JUXTAPID REMS Program, requiring certified healthcare providers and pharmacies for prescribing and distributing the medication. It is only indicated for patients with a clinical or laboratory diagnosis consistent with HoFH.2
References:
- Press release: Chiesi Global Rare Diseases Announces FDA Approval of JUXTAPID® (lomitapide) Capsules for Pediatric Use in Homozygous Familial Hypercholesterolemia (HoFH) https://chiesirarediseases.com/media/20251215chiesi-global-rare-diseases-announces-fda-approval-of-juxtapid-lomitapide-capsules-for-pediatric-use-in-homozygous-fa
Prescribing information: https://resources.chiesiusa.com/Juxtapid/JUXTAPID_PI.pdf