Lomitapide: A Medication Use Evaluation & a Formulary Perspective

Abstract Background and aims: Lomitapide is approved for lowering LDL-C in homozygous familial hypercholesterolemia, a rare genetic disorder. The evidence on its safety and efficacy from a small clinical trial requires further validation on effectiveness and safety in real-world. The aim of this study was to utilize institutional data on effectiveness and safety of lomitapide; to assist in formulating a perspective on adding it to the formulary. Methods: This was a retrospective review of patients that were actively being prescribed lomitapide at King Abdulaziz Medical City, Riyadh, Saudi Arabia from 2019 to 2022. Data collection included: demographics, confirmed gene mutation results, duration of lomitapide therapy, baseline, on treatment, and last LDL-C, percent reduction in LDL-C after 1-3 months of therapy (whichever was first available), other LDL-C lowering therapies used, liver function tests, adverse effects and compliance. Results: Eight adult patients were included in the review, the mean age was 25.5 years old, ~75% were female, and the duration of treatment with lomitapide ranged from 9 months to 3 years. None of the patients were on continuous LDL-apheresis. The mean baseline LDL-C at presentation to our facility was 17.2 mmol/L (range 11.78- 21.97 mmol/L), the mean percent drop in LDL-C with lomitapide was 34.1% (range 0- 87%), gastrointestinal disturbances were documented in 50% of the patients, and no cases of severe liver toxicities or increase in liver enzymes were seen. Conclusions: In our cohort of adult patients, lomitapide showed an overall modest reduction in LDL-C, with no cases of increase in liver enzymes and documented intolerance indicating that most patients were likely non-compliant. This review revealed important considerations when reimbursing expensive medications for rare diseases. Real-world evidence in real-time can support health care systems in price negotiations and reaching mutual agreements that eventually can improve patient access to care.