The US Food and Drug Administration (FDA) has approved Juxtapid (lomitapide) capsules for the treatment of pediatric patients ages 2 years and older with homozygous familial hypercholesterolemia (HoFH).
HoFH is a condition characterized by very high levels of cholesterol in the blood due to genetic changes in the LDLR gene. Patients with HoFH have a high risk of developing coronary artery disease, as well as health problems related to the buildup of excess cholesterol in other tissues. There are other hereditary forms of the condition caused by genetic changes in the APOB, LDLRAP1, or PCSK9 gene.
Lomitapide is a microsomal triglyceride transfer protein inhibitor designed to lower cholesterol associated with HoFH. It was first approved for the treatment of adults with HoFH in 2012.
The expanded indication approval is based on data from the phase 3, open-label, multicenter APH-19 (NCT04681170) clinical trial evaluating the safety and efficacy of lomitapide in 43 patients ages 5 to 17 years with HoFH. A 49% reduction from baseline in low-density lipoprotein cholesterol was observed. Significant decreases were also observed in non-high-density lipoprotein cholesterol, total cholesterol, very low-density lipoprotein cholesterol, apolipoprotein B, and triglycerides. Adverse events mainly consisted of gastrointestinal or hepatic issues, consistent with the known safety profile of lomitapide.
For more information, visit chiesirarediseases.com
To learn more about HoFH and other rare metabolic conditions, visit https://checkrare.com/diseases/metabolic-disorders/