ARIAD Pharma Announces FDA Full Approval And Label Update For Iclusig

(RTTNews) - ARIAD Pharmaceuticals Inc. (ARIA) announced that the U.S. Food and Drug Administration has granted Iclusig (ponatinib) full approval for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia or CML or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) for whom no other tyrosine kinase inhibitor (TKI) therapy is indicated; and for the treatment of adult patients with T315I-positive CML (chronic phase, accelerated phase, or blast phase) or T315I positive Ph+ ALL.

Iclusig was initially approved in December 2012 under the FDA's accelerated approval program, which provides patients earlier access to promising new drugs that treat serious conditions based on a surrogate endpoint while the company conducts additional studies to confirm the drug's clinical benefit. The therapy was granted the FDA's orphan drug designation because it is intended to treat a rare disease or condition.

The full approval and label update is based on 48-month follow-up data (as of August 2015) from the pivotal Phase 2 PACE clinical trial of Iclusig in heavily pretreated patients with resistant or intolerant CML or Ph+ ALL. These data were presented at the 2016 meetings of the American Society for Clinical Oncology and the European Hematology Association (EHA).

The efficacy and safety of ponatinib in CML and Ph+ ALL patients resistant or intolerant to dasatinib or nilotinib, or with the T315I mutation, were evaluated in the PACE trial. A total of 449 patients were treated with ponatinib at a starting dose of 45 mg/day. An estimated 93 percent of patients previously received two or more approved tyrosine kinase inhibitors (TKIs), and 56 percent of all patients had received three or more approved TKIs. Enrollment in the PACE trial was completed in October 2011.

Updated data on CP-CML patients (n=270) from the ongoing trial indicate that with a minimum follow-up of 48 months (data as of August 3, 2015), 110 patients continued to receive ponatinib. Additional data for CP-CML patients include: 55 percent of CP-CML patients achieved major cytogenetic response (MCyR) (primary endpoint) at any time; 39 percent of patients achieved a major molecular response (MMR) at any time; Six percent of all patients experienced a venous thromboembolic event.