Chiesi Global Rare Diseases has received a positive CHMP opinion for lomitapide in children with homozygous familial hypercholesterolemia, moving closer to an EU label expansion.

The Committee for Medicinal Products for Human Use of the European Medicines Agency recommended approval of lomitapide capsules for paediatric patients aged five years and older. A European Commission decision is expected by June 2026.

Lomitapide is already approved in the EU for adults with the rare genetic disorder, which is characterised by extremely high levels of low-density lipoprotein cholesterol and a high risk of early cardiovascular disease.

Homozygous familial hypercholesterolemia is an ultra-rare condition affecting an estimated one in 250,000 to one in 360,000 people worldwide. Without early treatment, patients face rapid progression of atherosclerosis and increased risk of premature death.

The CHMP opinion is based on data from the Phase 3 Aph-19 study, which evaluated lomitapide in 43 paediatric patients aged between five and 17 years. The study met its primary endpoint, showing a mean reduction of 53.5% in LDL cholesterol from baseline at week 24.

Reductions were also observed across multiple secondary lipid parameters, including total cholesterol, non-HDL cholesterol and triglycerides. The safety profile was consistent with previous studies, with most adverse events reported as mild and primarily gastrointestinal or hepatic.

One serious treatment-emergent adverse event was reported, related to elevated hepatic enzymes, which was managed through dose adjustment. No new safety signals were identified.

Mitch Goldman, svp research and development at Chiesi Global Rare Diseases, said: “Children living with HoFH deserve treatment options that meet their needs from the very start. The CHMP’s positive opinion brings us one step closer to offering lomitapide to those 5 years old and over.”

He added: “By expanding access to a therapy that has already benefited adult patients, we aim to help families navigate this condition with confidence and continuity of care.”

If approved, the decision would extend access to lomitapide to younger patients, addressing an unmet need in a population where treatment options remain limited and early intervention is critical.

The therapy is currently used alongside dietary management and other lipid-lowering treatments, including LDL apheresis, in adult patients. Expanding its use into paediatric populations would align with broader efforts to manage the disease earlier and reduce long-term cardiovascular risk.