PONATINIB APPROVED BY EMA

The European Commission has granted marketing authorization to ponatinib (Iclusig, Ariad Pharmaceuticals) as an orphan drug for use in certain leukemia patients who have stopped responding to or cannot tolerate other therapies.

Specifically, ponatinib is indicated for use in adults with chronic myeloid leukemia (CML) in the chronic, accelerated, or blast phase who are resistant to dasatinib (Sprycel) or nilotinib (Tasigna), who are intolerant of dasatinib or nilotinib and for whom subsequent treatment with imatinib (Gleevec) is not clinically appropriate, or who have the T315I mutation.

In addition, it is indicated for adults with Philadelphia (Ph) chromosome-positive acute lymphoblastic leukemia (ALL) who are resistant to dasatinib, who are intolerant to dasatinib and for whom subsequent treatment with imatinib is not clinically appropriate, or who have the T315I mutation.

Ponatinib was approved for similar indications in the United States in December 2012.

The manufacturer notes that ponatinib was granted accelerated assessment by the European Committee for Medicinal Products for Human Use. The aim of accelerated assessment is to expedite the review process for new medicines that address a major public health interest.

This approval in Europe "will offer CML patients, some of whom have run out of other treatment options, a new opportunity to improve their clinical outcome," said Stephen G. O'Brien, MD, PhD, professor of hematology at the Northern Institute for Cancer Research at Newcastle University in the United Kingdom, in a statement.

Other experts have also acknowledged the new hope offered by ponatinib.

"We have simply never had any treatment produce such high rates of durable response in a heavily pretreated group of patients," said Jorges Cortes, MD, professor of medicine and deputy chair of the Department of Leukemia at the University of Texas M.D. Anderson Cancer Center in Houston. He was commenting on preliminary results from the phase 2 PACE (Ponatinib Ph+ ALL and CML Evaluation) trial, which was the basis for the approval of the drug.

He presented the PACE results at the 2012 American Society of Hematology (ASH) annual meeting, where the trial was highlighted as "a major advance" by ASH president Armand Keating, MD, professor of medicine and director of hematology at the University of Toronto. Dr. Keating noted that ponatinib offers hope of a rescue medication for patients who have stopped responding to other therapies.

When results from a phase 1 clinical trial of ponatinib were published (N Engl J Med. 2012;367:2075-2088), they were described as "very encouraging" in an accompanying editorial by John M. Goldman, DM, FRCP, from Imperial College London, United Kingdom (N Engl J Med. 2012;367: 2148-2149). Dr. Goldman also wrote that ponatinib could be the "best of the bunch" of next-generation tyrosine kinase inhibitors for managing CML.