Advanced data modeling performed by researchers in the University of Florida College of Pharmacy has paved the way for a newly approved pediatric treatment for a rare and fatal genetic cholesterol disorder.
In March, the U.S. Food and Drug Administration approved the drug lomitapide for children over the age of 2 with homozygous familial hypercholesterolemia, or HoFH. The disease causes severely elevated cholesterol levels from birth and leads to heart attacks and cardiovascular disease during childhood or adolescence if left untreated. HoFH affects an estimated 1 in 170,000 to 1 in 300,000 individuals worldwide.
The approval was supported in part by pharmacokinetic and pharmacodynamic analyses conducted by faculty in the UF College of Pharmacy’s Department of Pharmaceutics and the Center for Pharmacometrics and Systems Pharmacology. The UF researchers developed model-informed dosing recommendations that demonstrated the medication could safely and effectively reduce harmful low-density lipoprotein, or LDL, cholesterol levels in pediatric patients.
Read more about UF Researchers Help Secure FDA Approval for Treatment Targeting Rare Pediatric Disease.
