Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy.

摘要: Duchenne muscular dystrophy (DMD) is an inherited neuromuscular disorder occurring in boys and caused by mutations the dystrophin gene. Vamorolone a first-generation delta-9,11 compound that has favorable efficacy side effect profiles relative to classical glucocorticoids. The pharmacokinetics (PK) of oral vamorolone were assessed parallel-group studies healthy men (phase 1, n = 86) with DMD 2a, 48) during 14 days once-daily dosing range doses. exhibited moderate variability PK, maximum plasma concentration usually at 2-4 hours half-life approximately 2 for all doses examined. Population PK modeling data together indicated can be well described 1-compartment model zero-order absorption. Both showed dose-linearity parameters examined, no accumulation drug daily dosing. Ingestion food resulted markedly enhanced absorption drug, as tested men. There similar apparent clearance averaging 2.0 L/h/kg 1.7 boys. Overall, well-behaved linear DMD, parameters, disposition those