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FDA approves imatinib mesylate (Gleevec) to treat pediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia

On September 27, 2006, the U. S. Food and Drug Administration granted accelerated approval to imatinib mesylate (Gleevec, Novartis Pharmaceuticals) as a single agent for the treatment of pediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukemia (Ph+ CML).

Approval is based upon the induction of both hematologic and cytogenetic responses.  A total of 51 pediatric patients with newly diagnosed and untreated chronic phase CML were enrolled in an open-label, multi-center, single arm phase 2 trial.  Patients were treated with Gleevec 340 mg/m2/day.  Complete hematologic response (CHR) was observed in 78% of evaluable pediatric patients after 8 weeks of therapy.  The complete cytogenetic response rate (CCyR) was 65%, comparable to the results observed in adult CML patients.  The partial cytogenetic response (PCyR) rate was 16%.  The majority of evaluable patients who achieved a CCyR developed the CCyR between months 3 and 10 (median time to response 6.74 months).  Estimated 12 month survival was 98% and estimated 24 month survival was 84%.

Imatinib generally was well tolerated.  Grade 3 or 4 toxicities were primarily hematologic.  Non-hematological grade 3 or 4 toxicities included allergic reaction/hyper-sensitivity, avascular osteonecrosis, and desquamating rash.  No deaths occurred on study therapy.  Only one patient discontinued study drug due to suspected study drug-related AEs (elevated AST/ALT).  Muscle cramps were reported sporadically during the study and there were no episodes of GI hemorrhage.  No new safety concerns were raised.

 A required phase 4 commitment is to continue follow-up of pediatric patients with Ph+ CML treated in the Phase 2 study to obtain long-duration safety and efficacy data.

Full prescribing information, including clinical trial information, safety, dosing, drug-drug interactions, and contraindications is available  in  Drugs@FDA

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System by phone at 1-800-FDA-1088, by facsimile 1-800-FDA-0178, by mail, or by using the Form 3500 


Page Last Updated: 12/29/2015
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