FDA

Juxtapid now approved for pediatric patients with homozygous familial hypercholesterolemia

March 9, 2026

On March 3, 2026, FDA approved Juxtapid (lomitapide) for pediatric patients ≥2 years of age with homozygous familial hypercholesterolemia (HoFH), as an adjunct to a low-fat diet and exercise and other low density lipoprotein cholesterol (LDL-C) therapies to reduce LDL-C.

Efficacy

A single-arm, open-label, 104-week trial (APH-19) evaluated the efficacy of Juxtapid when coadministered with a low-fat diet in 43 patients aged 5 to 17 years old with HoFH on stable lipid lowering therapy (LLT). The majority (98%) of study participants were White (one [2%] was Black or African American and one [2%] identified as Hispanic/Latino). Concomitant LLT at baseline included one or more of the following: statins (91%), ezetimibe (74%), and evolocumab (12%); 19 (44%) patients were receiving LDL apheresis through week 24.

A single‑arm study design was used because the disease is rare and assessing long‑term safety outcomes like growth and sexual maturation would not have been feasible in the context of a placebo‑controlled study. To offset the limitations of a single‑arm approach, the study included a 6‑week run‑in period to stabilize LLT and establish baseline levels, allowing each patient to serve as their own control.

The 6-week run-in was followed by a 24-week efficacy evaluation and an 80-week safety follow-up. During the efficacy phase, patients received an age‑dependent starting dose of Juxtapid, which was then increased to the maximum tolerated dose for their age group. The primary endpoint was percent change in LDL-C from baseline to week 24. Results showed the mean percent change in LDL-C from baseline to week 24 was -49% (95% CI: -59%, -38%). Subgroup analysis was carried out on patients aged 5 to 10 years (n=20) and 11 to 17 years (n=23). Mean decreases from baseline in LDL-C at week 24 were 52% and 46%, respectively.

The key secondary endpoint was the mean percent change in lipid parameters from baseline to week 24. Significant mean reductions were demonstrated for each of the lipid parameters assessed: total cholesterol (-45%), apolipoprotein B (-48%), triglycerides (-46%), non-high-density lipoprotein cholesterol (-49%), and very low-density lipoprotein cholesterol (-46%).

Safety

Most common adverse reactions in pediatric patients aged 5 to 17 years old (≥15%) were abdominal pain, increased ALT adn AST, diarrhea, and vomiting.

The prescribing information has a boxed warning for hepatotoxicity risk. Juxtapid is only available through the JUXTAPID REMS Program.

Recommended dose

The recommended starting dosage of Juxtapid is 2 mg orally once daily in patients aged 2 to 15 years old and 5 mg orally once daily in patients ≥16 years old. Escalate dose according to the titration schedule in the Juxtapid package insert, based on patient's age.

Sources:

Chiesi Global Rare Diseases. (2026, March 3). Chiesi Global Rare Diseases Announces FDA Approval of Juxtapid (lomitapide) Capsules for Pediatric Use in Homozygous Familial Hypercholesterolemia (HoFH). [Press release]. https://chiesirarediseases.com/media/20251215chiesi-global-rare-diseases-announces-fda-approval-of-juxtapid-lomitapide-capsules-for-pediatric-use-in-homozygous-fa

Juxtapid (lomitapide). [Package insert]. Chiesi Farmaceutici S.p.A. https://www.accessdata.fda.gov/drugsatfda_docs/label/2026/203858s027lbl.pdf Revised February 2026.