Rare Disease Report: FDA Fully Approves Iclusig for Chronic Myeloid Leukemia

November 30, 2016

(Source) The FDA has fully approved ARIAD Pharmaceuticals’ Iclusig (ponatinib) for the treatment of adult patients with chronic phase, accelerated phase, or blast phase chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Iclusig was approved in December 2012 under the FDA’s accelerated approval program.

The full approval is based on 48-month follow-up data from the Phase 2 trial. CML patients in the pivotal trial have retained long-term cytogenetic and molecular responses.

The kinase inhibitor was given a conditional approval in December, 2012.

Data was presented at the 2016 meetings of the American Society for Clinical Oncology (ASCO) and the European Hematology Association (EHA).

About the trial

449 patients diagnosed with CML and who were resistant or intolerant to dasatinib or nilotinib, or with the T315I mutation, were treated with Iclusig starting at a dose of 45 mg/day. 93% of treated patients previously received two or more approved tyrosine kinase inhibitors (TKIs), and 56% of all patients had received three or more approved TKIs.

During the 48-month follow up study, 55% of CP-CML patients achieved major cytogenetic response (MCyR). 39% of patients achieved a major molecular response (MMR).

About Iclusig

Iclusig is a kinase inhibitor with a target of BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia and Philadelphia-chromosome positive acute lymphoblastic leukemia. It targets BCR-ABL’s isoforms that carry mutations that confer resistance to treatment, including the T315I mutation.

Treatment of Iclusig is intended for patients whom no other tyrosine kinase inhibitor (TKI) therapy is indicated.

Better days for ARIAD

Last time ARIAD was in the media spotlight, it was because of former 2016 presidential candidate Bernie Sanders who sent a letter to the company, demanding more information about its price increases for Iclusig, which caused a stock price decrease. Good to see their latest news is a positive one and their stock is back up.

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