Two medicines have received positive opinions from an EU panel, potentially broadening treatment options for severe dyslipidemia and obesity in high-risk pediatric and adult populations.

At its March 2026 meeting, the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) issued positive opinions for Lojuxta (lomitapide, Chiesi Farmaceutici S.p.A.) and Imcivree (setmelanotide, Rhythm Pharmaceuticals Netherlands B.V.), addressing unmet needs in patients with refractory cholesterol levels and obesity driven by hypothalamic dysfunction.

If cleared by the European Commission, the decisions would give clinicians new options for two difficult-to-manage metabolic conditions.

Pediatric Breakthrough for Rare Hypercholesterolemia 

The first update concerns Lojuxta for homozygous familial hypercholesterolemia (HoFH). Previously authorized for adults, the CHMP has recommended extending its indication to include pediatric patients aged 5 years or older who remain at high cardiovascular risk despite standard therapies.

HoFH is a rare inherited disease characterized by extremely high blood levels of low-density lipoprotein (LDL) cholesterol. Genetic confirmation of HoFH should be obtained whenever possible, and other forms of primary hyperlipoproteinemia and secondary causes (eg, nephrotic syndrome, hypothyroidism) must be excluded.

Lojuxta is used as an adjunct to a low-fat diet and other lipid-lowering medicinal products for the treatment of HoFH. It contains lomitapide as active substance, which inhibits microsomal triglyceride transfer protein in the liver and gut, preventing the assembly and release of lipoproteins into the bloodstream.

The drug is an oral capsule taken once daily on an empty stomach, at least 2 hours after the evening meal.

Pediatric data from the phase 3 APH-19 trial in 43 patients aged 5-17 years with HoFH who received lomitapide showed a mean LDL cholesterol reduction of 53.5% (P <.0001) at week 24, with concordant decreases in non-high-density lipoprotein cholesterol (53.9%), total cholesterol (50.0%), apolipoprotein B (52.4%), very low-density lipoprotein cholesterol, and triglycerides.

Adverse events were gastrointestinal and hepatic; events of special interest occurred in 12% of patients, including one serious case of increased hepatic enzymes managed with dose interruption/reduction. Known risks include elevated hepatic transaminase levels and frequent gastrointestinal effects, and the drug is contraindicated in moderate to severe hepatic impairment and during pregnancy. 

Lojuxta was authorized under exceptional circumstances and treatment should be initiated and monitored by specialists, with ongoing risk minimization measures and hepatic monitoring.

Managing Acquired Hypothalamic Obesity 

The second recommendation involves Imcivree for the treatment of obesity and hunger control in adults and children aged 4 years or older with acquired hypothalamic obesity resulting from hypothalamic injury or impairment, often following the treatment of nonmalignant tumors leading to impaired melanocortin 4 receptor (MC4R) signaling and insatiable hunger. 

Imcivree contains setmelanotide, an MC4R agonist. It directly activates receptors in the brain to promote satiety and reduce excessive food intake in patients where the natural signaling pathway is impaired.

The drug is administered as a once-daily subcutaneous injection.

The recommendation was supported by evidence from a phase 2 multicenter trial in 18 patients aged 6-40 years with acquired hypothalamic obesity, in which 89% achieved at least a 5% BMI reduction at 16 weeks (P < .0001),. The mean BMI change was −15% and meaningful hunger score reductions were observed in patients aged 12 years or older. In a long-term extension, 12 of 14 patients who completed at least 12 months of the study had a mean BMI change of −26% from index baseline.

Common adverse events included nausea (61%), vomiting (33%), skin hyperpigmentation (33%), and diarrhea (22%).

Imcivree retains its existing indications for the treatment of obesity and hunger associated with genetically confirmed Bardet-Biedl syndrome and with biallelic loss-of-function POMC (including PCSK1) or LEPR deficiency. The full indication now specifies adults and children aged 2 years or older for these genetic forms.

Imcivree is designated as an orphan medicine and was granted entry to the EMA Priority Medicines scheme during its development. 

Both medications are subject to additional monitoring to further evaluate long-term safety and effectiveness through mandated postauthorization studies. 

For both newly recommended extensions, detailed recommendations for use will be provided in the updated Summary of Product Characteristics, to be published on the EMA website in all official European Union languages after the European Commission issues a decision (typically within approximately 67 days of the CHMP opinion).