EC nod for Chiesi Lojuxta for pediatric HoFH
Decision brief
Answer first · skim in under a minute
The European Commission has expanded Lojuxta's indication to include children aged 5 years and older with homozygous familial hypercholesterolemia. This analysis covers the regulatory shift, implications for Chiesi's rare disease franchise, and what investors and BD teams should monitor next.
The European Commission expanded Chiesi’s Lojuxta (lomitapide) to children aged 5 years and older with homozygous familial hypercholesterolaemia (HoFH). The June 2026 decision follows a March 2026 CHMP positive opinion and Phase 3 APH-19 data showing a mean 53.5% LDL-C reduction at week 24, with clear implications for rare-disease BD and EU market access.
Contents10 sections
Key Takeaways
- EC approval announced 5 June 2026 expands LOJUXTA to paediatric HoFH patients aged 5 years and older, alongside diet and other lipid-lowering therapies.
- CHMP adopted a positive opinion on 26 March 2026 recommending the adult-and-paediatric (5+) indication wording for EMEA/H/C/002578.
- APH-19 met its primary endpoint: mean 53.5% LDL-C reduction at week 24 (p<0.0001), with significant secondary lipid reductions.
- Competitive context: Evkeeza’s EU label covers adults and adolescents aged 12+, so Lojuxta’s age-5 floor targets a younger oral niche.
What did regulators decide on Lojuxta for pediatric HoFH?
Chiesi Global Rare Diseases reported on 5 June 2026 that the European Commission approved LOJUXTA (lomitapide) capsules for children 5 years of age and older with HoFH. The medicine remains an adjunct to a low-fat diet and other lipid-lowering treatments, including LDL-apheresis where available. Adult EU use dates to 2013.
Confirm the company disclosure on GlobeNewswire’s 5 June 2026 Chiesi release. The EC step followed EMA scientific advice rather than replacing it.
What did CHMP recommend before the EC decision?
On 26 March 2026, CHMP adopted a positive opinion recommending a change to Lojuxta’s marketing authorisation for Chiesi Farmaceutici S.p.A. The proposed indication covers adult and paediatric patients aged 5 years and older with HoFH, as an adjunct to a low-fat diet and other lipid-lowering medicinal products with or without LDL apheresis.
EMA’s variation page records opinion date 26/03/2026, product number EMEA/H/C/002578, and active substance lomitapide (ATC C10AX12). See the EMA Lojuxta variation / CHMP opinion summary and the March 2026 CHMP meeting highlights.
Which APH-19 efficacy numbers matter for BD models?
Per Chiesi’s GlobeNewswire disclosure, APH-19 achieved its primary endpoint with a mean 53.5% reduction in LDL-C from baseline at week 24 (p<0.0001). Significant reductions (all p<0.0001) were also reported for non-HDL-C, total cholesterol, VLDL-C, apolipoprotein B, and triglycerides at week 24.
- Primary endpoint: mean −53.5% LDL-C at week 24
- Statistical threshold reported: p<0.0001
- Adverse events of special interest: 5 of patients (12%)
- One serious treatment-emergent hepatic enzyme elevation event with dose interruptions/reductions
- Company conclusion: no new adverse-event signals versus the known adult profile
How does the pediatric expansion fit Chiesi’s rare-disease franchise?
Lojuxta is an oral microsomal triglyceride transfer protein (MTP) inhibitor pathway product for an ultra-rare genetic disorder that elevates LDL-C from birth. Expanding from adults-only EU use to age 5+ widens the addressable treated population in markets where paediatric HoFH management starts early to slow atherosclerosis.
EMA’s public assessment summary still frames the original EU authorisation (31 July 2013) under exceptional circumstances with intensive monitoring needs. Teams should assume hepatic and gastrointestinal risk management remains central to payer and clinician uptake even after the paediatric label change. Background on the authorised product sits on the EMA Lojuxta EPAR.
How does Lojuxta compare with Evkeeza in EU HoFH?
Competitive framing should stay label-accurate. EMA’s Evkeeza EPAR lists indication as an adjunct to diet and other LDL-C-lowering therapies for adult and adolescent patients aged 12 years and older with HoFH. That leaves ages 5–11 as a Lojuxta-specific EU oral window under the new EC decision, while older patients may see oral MTP inhibition versus intravenous ANGPTL3 blockade (evinacumab) trade-offs.
See the EMA Evkeeza EPAR indication wording. Head-to-head paediatric superiority claims are not established in the sources above and should not be modelled as proven.
What remains unproven for investors and market access?
Long-term cardiovascular outcome benefit in paediatric HoFH is not settled by the week-24 LDL-C surrogate alone. Country-level HTA and pricing in Germany, France, Italy, Spain, and the UK will determine real revenue, not the EC decision text. US paediatric positioning (Juxtapid) is a separate regulatory story and should not be conflated with the EU SmPC without checking FDA labelling.
Unverified M&A narratives around Chiesi’s broader rare-disease deal flow are omitted here because they are not required to explain the Lojuxta paediatric decision.
Related NovaPharma coverage
- Regulatory round-up: 2 February 2026 – EMA opinions, FDA approvals
- New EU Template for Clinical Trial Recruitment and Informed Consent
- Health News Round Up: Diabetes, Vaping, and Biologics
Frequently Asked Questions
What did the European Commission approve for Lojuxta?
On 5 June 2026, Chiesi announced that the European Commission approved LOJUXTA (lomitapide) capsules for children 5 years of age and older with homozygous familial hypercholesterolaemia (HoFH), used with diet and other lipid-lowering treatments, including LDL-apheresis where available.
What evidence supported the pediatric indication?
The Phase 3 APH-19 study met its primary endpoint with a mean 53.5% reduction in LDL-C from baseline at week 24 (p<0.0001). Secondary lipid endpoints also improved significantly, and no new adverse-event signals were identified versus the known adult profile.
How does Lojuxta’s EU pediatric age cut-off compare with Evkeeza?
EMA’s Evkeeza (evinacumab) EPAR lists use as an adjunct for adult and adolescent patients aged 12 years and older with HoFH. Lojuxta’s EC pediatric expansion, per Chiesi’s June 2026 announcement, reaches down to age 5, creating a younger oral option alongside injectable ANGPTL3 blockade in older patients.
Primary Sources
Regulatory catalyst tracker
Track PDUFA dates, approval milestones, and label updates for Lojuxta.
Unlock full calendar →Chiesi pipeline snapshot
One-screen view of active programs, phases, and recent catalysts from public sources.
Investor brief
Download a one-page summary of regulatory impact and competitive context.
Explore drug hub →Entity graph
Continue Exploring
Open the drugs, companies, and topics behind this story.
Sources & references 1 primary sources
Sources verified at publication. See our editorial policy and data sources.
This article follows our editorial standards. Report a correction via editorial contact.
Deeper reading
Industry reports & whitepapers
- FDA CBER Patient Listening Meeting on Gene Therapy Safety: Patient and Care Partner Perspectives — Patient and care partner perspectives inform FDA’s thinking on approved gene therapy safety and long…