In collaboration with the Food and Drug Administration (FDA), and as a service to our members, ASH provides information about newly approved therapies for patients. This allows the agency to inform hematologists and professionals in hematology-related fields of recent approvals in a timely manner. Included in the message below is a link to the product label, which provides the relevant clinical information on the indication, contraindications, dosing, and safety. In providing this information, ASH does not endorse any product or therapy and does not take any position on the safety or efficacy of the product or therapy described. The following is a message from the FDA’s Office of Hematology and Oncology Products:
On December 14, 2012, the U. S. Food and Drug Administration granted accelerated approval to ponatinib (Iclusig™ tablets, ARIAD Pharmaceuticals, Inc.) for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy or Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ALL) that is resistant or intolerant to prior tyrosine kinase inhibitor therapy.
The approval was based on the results of the PACE trial, a multicenter, international, single-arm clinical trial of 449 patients with disease that was resistant or intolerant to prior tyrosine kinase inhibitor therapy.The primary endpoints were Major Cytogenetic Response (MCyR) for patients with CP-CML and Major Hematologic Response (MaHR) for patients with AP-CML, BP-CML or Ph+ALL. FDA required that the sponsor commit to submit 24-month follow-up data for all patients as a condition for the accelerated approval.
The efficacy results demonstrated a 54% MCyR rate in patients with CP-CML. Seventy percent of patients with CP-CML with T315I mutation achieved MCyR. The median duration of MCyR had not yet been reached at the time of analysis. For patients with AP-CML, BP-CML and Ph+ ALL, the MaHR rates were 52%, 31% and 41%, respectively. The median duration of MaHR in patients with AP-CML, BP-CML and Ph+ ALL was 9.5 months, 4.7 months and 3.2 months respectively.
Iclusig is being approved with a Boxed Warning alerting patients and healthcare professionals that arterial thrombosis and liver toxicity have occurred in Iclusig-treated patients. The most common side effects reported in the clinical trial include hypertension, rash, abdominal pain, fatigue, headache, dry skin, constipation, fever, joint pain, and nausea.
The recommended dose and schedule for ponatinib is 45 mg taken orally once daily with or without food.
Full prescribing information is available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2012/203469lbl.pdf
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 completing a form online at http://www.fda.gov/medwatch/report.htm, by faxing (1-800-FDA-0178) or mailing the postage-paid address form provided online, or by telephone (1-800-FDA-1088).