Efficacy of Ponatinib

Ponatinib (Ponatinib) is an orally active polytyrosine kinase inhibitor currently approved by the U.S. Food and Drug Administration (FDA) for patients with chronic myelogenous leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia, specifically targeting the BCR-ABL gene mutation T315I. Due to the unique multi-target properties of ponatinib, further studies demonstrated its ability to target other important tyrosine kinases (FGFR, PDGFR, SRC, RET, KIT, and FLT1) in other human malignancies.

The first preclinical trial of ponatinib was reported in 2006, which proved the effective anti-BCR-ABL effect of ponatinib. These in vitro studies utilized Ba/F3 cell lines expressing native BCR-ABL or BCR-ABL with mutations within the tyrosine KD. Kinase selectivity studies found that ponatinib effectively blocked wild-type and several mutant forms of BCR-ABL kinase, including BCR-ABLT315I. In addition, this study compared the efficacy of several concentrations of ponatinib on cells from patients with primary leukemia blast blasts driven specifically by wild-type BCR-ABL or BCR-ABLT315I and monocytes from healthy individuals. Experimental data showed that ponatinib induced a specific reduction in viable cells relative to primary CML cells, showing an IC50 value approximately 500-fold lower than that of normal cells, confirming its potent pan-BCR-ABL inhibitory effect.

The original drug of Ponatinib has not yet been launched in China, so it is not included in the medical insurance. Domestic patients in need may only purchase it through overseas channels. The Hong Kong original version of Ponatinib specificationsThe price of 45mg*30 tablets per box may be around 40,000 yuan (the price may fluctuate due to the exchange rate), which is expensive. There are also generic ponatinib drugs produced in other countries, which are relatively cheap. The price of 45mg*30 tablets per box produced by Everest Pharmaceutical Factory in Bangladesh may be around 4,000 yuan (the price may fluctuate due to the exchange rate), and its drug ingredients are basically the same as those of the original drug.