Additional Relevant Details The NDA for ponatinib is supported by the Phase 1/2 study in Japanese patients, as well as the global pivotal PACE and Phase 1 trials of ponatinib. The Japanese study included 35 Japanese patients with CML or Ph+ALL who experienced failure of prior tyrosine kinase inhibitor (TKI) therapy. With a median follow-up of 14.9 months at the time of analysis, the Phase 1/2 study results confirm that Iclusig demonstrates anti-leukemic activity in this patient population. Among chronic-phase patients, 65% achieved the primary efficacy endpoint of major cytogenetic response, and 35% had a major molecular response. Thirteen patients remained on therapy including 12 in chronic phase. The median duration of response had not been reached. The most common treatment-emergent adverse events in the trial of any grade were decreased platelet and neutrophil counts, pyrexia, hypertension, dry skin, rash and increased lipase. Treatment-emergent arterial thrombotic events were reported in four patients, or 11%. According to a 2011 survey by the Ministry of Health Labor and Welfare in Japan, CML occurs in approximately 1 out of 100,000 people among all age groups and it is estimated that there are approximately 11,000 patients living with CML in Japan.