BOSTON, March 3, 2026 — Leads & Copy —
Chiesi Global Rare Diseases, a Chiesi Group business unit, announced that the U.S. Food and Drug Administration (FDA) has approved JUXTAPID® (lomitapide) capsules for pediatric use in children 2 years of age and older with homozygous familial hypercholesterolemia (HoFH).
The announcement was made during Rare Disease Day week, which raises awareness about rare diseases’ impact on patients.
JUXTAPID, used with diet and other lipid-lowering treatments, has been approved in the United States for adult patients with HoFH since 2012.
HoFH is an ultra-rare genetic disorder affecting an estimated 1 in 250,000 to 1 in 360,000 individuals worldwide, including children, who can be born with dangerously high low-density lipoprotein cholesterol (LDL-C) levels. Without early intervention, those living with this condition face a significantly increased risk of severe atherosclerosis.
Mitch Goldman, Senior Vice President, R&D, Chiesi Global Rare Diseases, said the approval represents a meaningful advancement for children and families living with HoFH. He added that expanding access to JUXTAPID for children 2 years of age and older enables young members of the HoFH community to benefit from the same proven treatment that has already helped adults manage their condition.
Katherine Wilemon, Founder and CEO of Family Heart Foundation, said that children with HoFH face extraordinary challenges from the moment they’re diagnosed. She added that the recent treatment approval for this age group marks a meaningful step forward for young children impacted by HoFH.
The FDA’s approval is based on evidence from a Phase 3, open-label, multicenter study (APH-19) evaluating the safety and efficacy of JUXTAPID® in 43 pediatric participants aged 5 to 17 years with HoFH who were receiving standard-of-care lipid-lowering therapy and a low-fat diet. Over a 24-week treatment period, JUXTAPID was titrated to each participant’s highest tolerated dose. The study demonstrated a mean 49% reduction in low-density lipoprotein cholesterol (LDL-C) from baseline, accompanied by significant decreases in non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol, very low-density lipoprotein cholesterol (VLDL-C), apolipoprotein B, and triglycerides. Adverse events were primarily gastrointestinal or hepatic in nature, consistent with the known safety profile of JUXTAPID. An integrated model-based analysis confirmed that JUXTAPID is safe and efficacious for the treatment of HoFH in pediatric populations and support the dose justification in children > 2 yrs old. Overall, the results showed that JUXTAPID achieved substantial, clinically meaningful reductions in LDL-C in pediatric use for HoFH.
JUXTAPID (lomitapide) is a prescription medicine used along with a low-fat diet, exercise and other low-density lipoprotein (LDL) lowering medicines to reduce LDL-C in adults and children 2 years of age and older with a type of high cholesterol called homozygous familial hypercholesterolemia (HoFH).
It is not known if JUXTAPID is safe and effective in people with kidney problems including people with end-stage kidney disease who are not on dialysis.
It is not known if JUXTAPID is safe and effective when used in children under the age of 2.
JUXTAPID is available only through certified pharmacies that are enrolled in the JUXTAPID REMS Program. A healthcare provider must be enrolled in the program to prescribe JUXTAPID.
There is a registry that collects information about the effects of taking JUXTAPID over time. For more information about this registry visit www.JUXTAPID.com or call 1-877-902-4099.
Do not take JUXTAPID if you are pregnant, think you may be pregnant, or are planning to become pregnant.
Chiesi is a research-oriented international biopharmaceutical group that develops and markets innovative therapeutic solutions in respiratory health, rare diseases, and specialty care. The company’s mission is to improve people’s quality of life and act responsibly towards both the community and the environment.
Chiesi is headquartered in Parma (Italy), with 31 affiliates worldwide, and counts more than 7,500 employees.
For more information visit www.chiesi.com.
Chiesi Global Rare Diseases is a business unit of the Chiesi Group established to deliver innovative therapies and solutions for people living with rare diseases.
For more information visit www.chiesirarediseases.com.
Follow @ChiesiGlobalRareDiseases on LinkedIn, Facebook, Instagram, X and YouTube.
Source: Chiesi Group
