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Ponatinib is a small molecule tyrosine kinase inhibitor used primarily for the treatment of chronic myeloid leukemia (CML) and Philadelphia chromosome–positive acute lymphoblastic leukemia (Ph+ ALL), especially in patients who are resistant or intolerant to prior therapies. It is particularly effective against CML with the T315I mutation, which confers resistance to other BCR-ABL inhibitors. Ponatinib works by inhibiting multiple kinases, most notably BCR-ABL (including T315I mutant), as well as VEGFR, PDGFR, FGFR, EPH receptors, SRC family kinases, KIT, RET, TIE2, and FLT3. The drug was developed by Ariad Pharmaceuticals and is marketed under the brand name Iclusig.
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