Interim analysis shows lomitapide safe and effective in Japanese patients with HoFH

Despite the occurrence of drug-related adverse events, including liver-related issues and gastrointestinal disorders, lomitapide demonstrated effectiveness in reducing LDL-C levels in Japanese patients with homozygous familial hypercholesterolemia.

The study suggests that lomitapide is safe and effective in real-world clinical practice in Japan, highlighting its potential as a treatment option for this patient population.

The study, which included 39 patients, examined the use of lomitapide over a period of 42 months. The median daily dose of lomitapide administered was 9.8 mg.

Results found that 74 drug-related adverse events were reported in 61.5% of patients. These adverse events included liver-related issues in 35.9% of patients, gastrointestinal disorders in 48.7%, and 1 case of a bleeding disorder. In response to these events, the dose of lomitapide was reduced for 39.2% of cases, temporarily withdrawn for 12.2%, and discontinued in only 1 instance.

Despite these adverse events, the study found that lomitapide demonstrated effectiveness in reducing LDL-C levels. The mean LDL-C level decreased from 225.9 mg/dl (5.8 mmol/l) before treatment to 159.4 mg/dl (4.1 mmol/l) after 12 months of treatment (P = 0.0245).

Reference
Harada-Shiba M, Haruna S, Kogawa N. Real-world safety and efficacy of lomitapide in homozygous familial hypercholesterolemia: interim report of special-use survey in Japan. Future Cardiol. 2024;doi: 10.2217/fca-2023-0136. Epub ahead of print. PMID: 38420884.