EC expands Lojuxta label to children with HoFH

Chiesi’s Lojuxta has secured a European Commission label expansion for pediatric use in homozygous familial hypercholesterolaemia, giving clinicians in the EU a newly approved option for children ages 5 and older with one of the most severe inherited lipid disorders. The decision follows the EMA’s Committee for Medicinal Products for Human Use, or CHMP, which adopted a positive opinion on March 26, 2026, recommending that the existing adult indication be broadened to include pediatric patients. (ema.europa.eu)

That’s a meaningful regulatory step for a product that has been on the EU market since 2013, when Lojuxta was first authorized for adults with HoFH. HoFH is an ultra-rare condition marked by very high LDL cholesterol from birth and a sharply elevated risk of early atherosclerotic cardiovascular disease. The 2023 European Atherosclerosis Society consensus update described HoFH as life-threatening, often diagnosed late, and still undertreated, while also emphasizing that treatment in children needs to begin early and usually requires combination therapy beyond standard statins and ezetimibe. (ema.europa.eu)

The newly expanded indication keeps Lojuxta positioned as an adjunct to a low-fat diet and other lipid-lowering medicinal products, with or without LDL apheresis. EMA’s variation notice says genetic confirmation of HoFH should be obtained whenever possible, and that other primary hyperlipoproteinaemias and secondary causes, including nephrotic syndrome and hypothyroidism, should be excluded. The product is marketed by Chiesi Farmaceutici S.p.A., and the pediatric pathway also included a modified paediatric investigation plan accepted by EMA in October 2024. (ema.europa.eu)

Chiesi’s earlier announcements provide more detail on the evidence package behind the expansion. In March, the company said the FDA had already approved pediatric JUXTAPID in the US, based on the Phase 3 APH-19 study in 43 pediatric participants ages 5 to 17 who were receiving standard lipid-lowering therapy and a low-fat diet. According to that release, lomitapide delivered a mean 49% reduction in LDL-C over 24 weeks, with adverse events mainly gastrointestinal or hepatic and consistent with the drug’s established profile. The Pediatric Endocrine Society’s drug update echoed those study basics and highlighted the diagnostic criteria used in the trial population. (globenewswire.com)

Industry and advocacy reaction has been predictably supportive. In Chiesi’s US announcement, Family Heart Foundation CEO Katherine Wilemon said children with HoFH face major cardiovascular risk from the time of diagnosis and called pediatric access to treatment a meaningful step forward. Company statements have also framed the label expansion as part of a broader rare-disease strategy, noting that lomitapide is reimbursed in 16 countries and has already received pediatric approval in Saudi Arabia for children ages 5 and older. Those comments should be read as stakeholder perspective rather than independent assessment, but they do reflect the longstanding pressure to widen access in a very small patient group with limited options. (globenewswire.com)

Why it matters: For veterinary professionals, this story is less about direct clinical use and more about how regulators are handling precision therapies for rare, inherited disease in pediatric populations. The Lojuxta decision shows the EU is willing to extend labels when a small but coherent data package supports benefit in children, even for drugs that require intensive monitoring. That’s notable because lomitapide has long been associated with liver toxicity risk, gastrointestinal adverse effects, drug interaction concerns, and pregnancy precautions, and EMA continues to list it under additional monitoring. In practical terms, it’s another example of the growing expectation that high-need pediatric populations shouldn’t wait for perfect data when the disease burden is immediate and severe. (ema.europa.eu)

There’s also a broader signal here for specialists who follow translational medicine and orphan-drug regulation. The 2023 EAS consensus update notes that therapies such as lomitapide act independently of LDL receptor function, which matters in HoFH because response to conventional LDL-lowering drugs can be limited by the underlying genetics. That mechanistic distinction helps explain why regulators and clinicians continue to view lomitapide as an important option despite its safety complexity. (academic.oup.com)

What to watch: The next milestones are the publication of the updated EU SmPC in all official languages, country-by-country reimbursement and access decisions, and fuller public reporting of longer-term pediatric data beyond the initial APH-19 efficacy readout. Those details will determine how quickly this approval translates into real-world use. (ema.europa.eu)