Chiesi Global Rare Diseases recently announced that the European Commission (EC) has approved lomitapide capsules for use in children 5 years of age and older with Homozygous Familial Hypercholesterolaemia (HoFH), for use alongside diet and other lipid-lowering treatments, including LDL-apheresis where available.

The EC decision follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommending the expanded indication. Lomitapide has been approved for use in the European Union (EU) for adult patients with HoFH since 2013.

Mitch Goldman, MD, PhD, SVP—Research and Development, Chiesi Global Rare Diseases, said, "This European Commission approval marks an important milestone for children living with HoFH and their families. HoFH is a condition that can begin causing irreversible cardiovascular damage from the earliest years of life, and the unmet need remains for younger patients despite existing treatment options. By expanding the indication of lomitapide to children aged 5 years and older, we are offering families and their physicians a meaningful treatment option that is backed by robust clinical evidence. This approval reflects our deepest commitment: ensuring that having a rare disease never means being overlooked at any stage of life, across generations.”

The EC approval is based on evidence from a phase 3, open-label, single-arm, multicenter study evaluating lomitapide in 43 pediatric participants aged 5 to 17 years with HoFH. The APH-19 study achieved its primary endpoint, demonstrating a mean 53.5 percent reduction in LDL?C from baseline at week 24 (p<0.0001). Significant reductions (all p<0.0001) were also achieved in non?HDL?C, total cholesterol, VLDL?C, apolipoprotein B, and triglycerides at week 24 (secondary outcomes).

Adverse Events (AEs) were mostly mild, and gastrointestinal and hepatic in nature. AEs of special interest were reported for 5 (12 percent) patients (gastrointestinal in 2 patients and hepatic in three).

One serious treatment-emergent AE was reported (also classed as an AE of special interest): an increase in hepatic enzymes, resulting in two dose interruptions, two dose reductions, and a repeated dose escalation. Overall, no new adverse event signals were identified, and the results were consistent with the known profile of lomitapide.