[4] In March 2024, the FDA expanded the indication to include the treatment, with chemotherapy, for adults with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia.

[12] Recently, an analogue of ponatinib was developed that retained anti-tumor efficacy but had reduced cardiovascular toxicity in experimental models.

[citation needed] The PACE (Ponatinib Ph+ ALL and chronic myeloid leukemia Evaluation) pivotal phase II trial started enrolling patients in September 2010 and is designed to provide definitive clinical data for regulatory approval in this setting.

[14][15] The EPIC (Evaluation of Ponatinib versus Imatinib in chronic myeloid leukemia) phase-III trial began in June 2012 [16] and was halted[clarification needed][11] on October 18, 2013.

Chronic myeloid leukemia is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein.

[20] NICE estimated that ponatinib would cost approximately £61,000 per year, but the price paid under the Cancer Drugs Fund is confidential and may be different.