Long-term study confirms effectiveness of lomitapide in HoFH

Lomitapide provides significant and sustained reductions in low-density lipoprotein cholesterol (LDL-C) levels in patients with homozygous familial hypercholesterolemia (HoFH), improving cardiovascular symptoms with manageable side effects over long-term treatment, according to a long-term study.

The study included patients with limited success with prior PCSK9 inhibitor treatment who transitioned to lomitapide. Patients were treated for a median duration of 47 months.

Treatment with lomitapide resulted in an 80% reduction in LDL-C levels at the lowest point and a 69% decrease at the final follow-up. Notably, 4 of the 6 patients achieved LDL-C levels below 70 mg/dL during the course of therapy.

Mild to moderate side effects were observed, with hepatic steatosis remaining manageable and liver elasticity normal in most patients. Cardiovascular symptoms improved across the cohort, with participants reporting stabilization or regression of arterial plaques.

Ongoing follow-up with larger patient groups is recommended to further explore cardiovascular benefits and safety.

Reference
Suppressa P, Coppola C, Cocco V, et al. Long-term effectiveness and safety of lomitapide in patients with homozygous familial hypercholesterolemia: an observational case series. Orphanet J Rare Dis. 2024;19(1):370. doi: 10.1186/s13023-024-03374-9. PMID: 39380044; PMCID: PMC11459886.