PARMA, Italy, June 5, 2026
Chiesi Global Rare Diseases has announced a major regulatory milestone following the European Commission’s approval of LOJUXTA® (lomitapide) capsules for children aged five years and older with Homozygous Familial Hypercholesterolaemia (HoFH), an ultra-rare inherited disorder characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C). The decision significantly expands treatment options for pediatric patients living with one of the most severe genetic cholesterol disorders and reinforces Chiesi’s commitment to advancing therapies for rare diseases. The approval enables LOJUXTA to be prescribed alongside dietary management and other lipid-lowering therapies, including LDL-apheresis where available, offering physicians an additional therapeutic tool to address the substantial cardiovascular risks associated with HoFH. The regulatory milestone follows a positive recommendation from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) and marks a new chapter in the management of pediatric rare cardiovascular diseases.
European Commission Expands Access for Young HoFH Patients
The European Commission approval extends the indication of LOJUXTA® (lomitapide) beyond adult patients to include children from the age of five, addressing a critical unmet medical need in a patient population vulnerable to early cardiovascular complications. HoFH is a rare genetic disorder that causes dangerously high cholesterol levels from birth, often leading to premature atherosclerosis, cardiovascular disease, and life-threatening cardiac events at a young age. Patients with HoFH can experience LDL cholesterol levels that are more than ten times higher than recommended targets, making early diagnosis and aggressive treatment essential.
Despite available therapies, many young patients remain undertreated, increasing the risk of irreversible cardiovascular damage. The expanded approval provides healthcare professionals with a valuable therapeutic option designed to help manage cholesterol levels earlier in life and improve long-term health outcomes for affected children.
Phase 3 Study Demonstrates Significant LDL Cholesterol Reduction
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The approval was supported by data from the Phase 3 APH-19 clinical trial, an open-label, multicenter study evaluating LOJUXTA in 43 pediatric patients aged 5 to 17 years diagnosed with HoFH. Results demonstrated a highly significant 53.5% mean reduction in LDL cholesterol levels from baseline at Week 24, successfully achieving the study’s primary endpoint. Investigators also reported substantial improvements across multiple secondary lipid parameters, including non-HDL cholesterol, total cholesterol, very-low-density lipoprotein cholesterol (VLDL-C), apolipoprotein B, and triglycerides.
These findings highlight the potential of lomitapide to deliver meaningful lipid-lowering benefits in a pediatric population facing lifelong cardiovascular risk. Importantly, the overall safety profile observed in the study remained consistent with previously established clinical experience, supporting the use of the therapy in younger patients. The strong efficacy outcomes underscore the role of targeted rare disease therapies in transforming care for genetically driven disorders.
Strengthening Rare Disease Innovation in Cardiovascular Care
The latest approval further strengthens Chiesi Global Rare Diseases’ position in the development of innovative treatments for underserved patient populations. By extending access to LOJUXTA for pediatric use, the company aims to help address one of the most challenging rare lipid disorders while supporting earlier intervention strategies designed to prevent long-term cardiovascular complications. As precision medicine and rare disease research continue to evolve, regulatory approvals such as this demonstrate the growing importance of targeted therapies backed by robust clinical evidence.
The decision also reflects increasing recognition among regulators and healthcare providers that rare pediatric diseases require dedicated treatment pathways to improve quality of life and clinical outcomes. With LOJUXTA now approved for both adult and pediatric HoFH patients across the European Union, Chiesi continues to expand its rare disease portfolio and reinforce its commitment to delivering innovative solutions for patients with significant unmet medical needs.
Source: Chiesi Global Rare Diseases press release
