Chiesi Global Rare Diseases, a division of the Chiesi Group, has announced a breakthrough: the US FDA has approved JUXTAPID (lomitapide) capsules for pediatric use in kids with ultra-rare genetic cholesterol disorder.
Lomitapide, a therapy already approved for adults since 2012, is now set to help the youngest patients manage dangerously high cholesterol levels caused by homozygous familial hypercholesterolemia (HoFH). It affects roughly 1 in 250,000 to 1 in 360,000 people worldwide. Left untreated, HoFH dramatically increases the risk of severe atherosclerosis.
“This approval represents more than a regulatory milestone; it’s a meaningful advancement for children and families living with HoFH,” said Mitch Goldman, Senior Vice President, R&D, Chiesi Global Rare Diseases.
"By expanding access to lomitapide 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. This achievement reflects our unwavering commitment to bringing therapies to those who need them most and to making a lasting difference at every stage of life.”
Katherine Wilemon, Founder and CEO of Family Heart Foundation, added: “Children with HoFH face extraordinary challenges from the moment they’re diagnosed. Their lives are shaped by frequent medical visits and the constant worry of cardiovascular risk at an age when most kids are just learning to ride a bike or play sports. The recent treatment approval for this age group marks a meaningful step forward for young children impacted by HoFH.”
The FDA’s decision follows results from a Phase 3 study involving 43 pediatric participants aged 5 to 17. Over 24 weeks, lomitapide, administered alongside standard-of-care lipid-lowering therapy and a low-fat diet, achieved a mean 53.5% reduction in LDL cholesterol, alongside decreases in total cholesterol, VLDL-C, apolipoprotein B, triglycerides, and non-HDL cholesterol. Side effects were mainly gastrointestinal or hepatic, consistent with the known safety profile.
