Therapeutic efficacy of Ponatinib

Chronic Phase (CP) CML

The efficacy of Iclusig was evaluated in OPTIC (NCT02467270), a dose-optimization trial. Eligible patients had CP-CML whose disease was considered to be resistant or resistant/intolerant to at least  2 prior kinase inhibitors or who have the T315I mutation.

Of the 45 patients who had a dose reduction after achieving ≤1% BCR-ABL1IS, 28 patients (62%)  maintained their response at the reduced dose for at least 90 days. Of these 28 patients, 18 patients  (64%) maintained the response for at least one year. Median duration of response (MR2) was not  reached.

Chronic Phase (CP), Accelerated Phase (AP), Blast Phase (BP) CML and Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)

The efficacy of Iclusig was evaluated in PACE (NCT01207440), a single-arm, open-label,  international, multicenter trial.

In patients with CP-CML who achieved MCyR or MMR, the median time to response was 3 months  (range: 1.8 to 12.3 months) and 6 months (range: 2 to 60.2 months), respectively. With a minimum  follow-up of 60 months, the median durations of MCyR (range: 1 day to 70.1 months) and MMR  (range: 1 day to 67.8 months) had not yet been reached.

FDA,2022.02