Lomitapide

Lomitapide is a potent (IC50 = 8 nM) inhibitor of microsomal triglyceride transfer protein (MTP or MTTP) and is clinically useful in treating familial hypercholesterolemia.1,2 It has displayed anticancer activity acting through various pathways including autophagy-dependent cancer cell death via direct inhibition of the kinase activity of mTORC13, activation of AMPK/Beclin1-mediated autophagy4, Notch inhibition via targeting TACE and γ-secretase5, blocking ZDHHC5-dependent palmitoylation on SSTR56, PFKFB3 inhibition7, and PARP14/DRP1-mediated mitophagy8.