FDA approves lomitapide for children aged 2 years and older with HoFH

The FDA has approved lomitapide (JUXTAPID; Chiesi) capsules for children aged 2 years and older with homozygous familial hypercholesterolemia (HoFH), according to Chiesi Global Rare Diseases.¹

This approval was based on positive phase 3 safety and efficacy data from a multicenter study of pediatric patients aged 5 to 17 years with HoFH. Before the intervention, these patients were undergoing standard care through lipid-lowering therapy and a low-fat diet. After switching to the intervention, significant improvements were observed.

“By expanding access to JUXTAPID for children 2 years of age and older, we’re enabling very young members of the HoFH community to benefit from the same proven treatment that has already helped adults manage their condition,” said Mitch Goldman, senior vice president of R&D at Chiesi Global Rare Diseases.

Significant reductions and safety profile

The improvements observed from lomitapide included a 49% reduction in low-density lipoprotein cholesterol (LDL-C) from baseline to week 24. Additionally, significant declines were observed in total cholesterol, triglycerides, non-high-density lipoprotein cholesterol, very low-density lipoprotein cholesterol, and apolipoprotein B.

A consistent safety profile was noted with previous research for lomitapide, with most adverse events being gastrointestinal or hepatic in nature. Based on this safety data and the clinically meaningful reductions observed in LDL-C, investigators concluded lomitapide is safe and effective for use in pediatric patients.

Lomitapide is designed to be used alongside a low-fat diet, exercise, and other low-density lipoprotein lowering medications. These strategies can reduce LDL-C in both pediatric and adult HoFH patients.

Imapct of HoFH

HoFH is estimated in 1 in 250,000 to 360,000 individuals. These include children who may be born with dangerously high LDL-C levels, facing a significant increase in severe atherosclerosis risk if treatment is not provided early.

“Children with HoFH face extraordinary challenges from the moment they’re diagnosed,” said Katherine Wilemon, Founder and CEO of Family Heart Foundation. “Their lives are shaped by frequent medical visits and the constant worry of cardiovascular risk... The recent treatment approval for this age group marks a meaningful step forward for young children impacted by HoFH.”

Renewed emphasis on pediatric screening

The approval comes at a time when major organizations are calling for a renewed emphasis on screening and management for familial hypechoelsterolemias (FH) among pediatric patients.² Less than 2 months before the approval in January 2026, the National Lipid Association (NLA) released an updated consensus on the topic.

In this document, the NLA called for screening for suspected HoFH to occur as soon as the risk is recognized, ideally before the age 2 years, while universal screening for all children is recommended between ages 9 and 11 years.

Screening should include a thorough review of medical and family history, physical examination, and comprehensive laboratory examination. FH may be diagnosed when the relevant clinical characteristics are present and secondary causes and phenocopies have been ruled out. The severity of LDL-C is also considered.

“Genetic testing is not required to establish a diagnosis of FH, but such testing is appropriate to complement the clinical diagnosis and facilitate cascade screening,” wrote NLA authors.

References

1. Chiesi Global Rare Diseases announces FDA approval of JUXTAPID® (lomitapide) capsules for pediatric use in homozygous familial hypercholesterolemia (HoFH). Chisei Global Rare Diseases. March 3, 2026. Accessed March 6, 2026. https://chiesirarediseases.com/media/20251215chiesi-global-rare-diseases-announces-fda-approval-of-juxtapid-lomitapide-capsules-for-pediatric-use-in-homozygous-fa
2. Ahmad Z, Agarwala A, Cuchel M, et al. Update on familial hypercholesterolemia: An expert clinical consensus from the National Lipid Association. Journal of Clinical Lipidology. 2026. doi:10.1016/j.jacl.2026.01.011