Drugs Xagena
The U.S. Food and Drug Administration ( FDA ) has granted accelerated approval of Iclusig ( Ponatinib ) for the treatment of adult patients with chronic, accelerated or blast phase chronic myeloid leukemia ( CML ) that is resistant or intolerant to prior tyrosine kinase inhibitor ( TKI ) therapy or Philadelphia chromosome-positive acute lymphoblastic leukemia ( Ph+ ALL ) that is resistant or intolerant to prior TKI therapy.
Approximately 5,000 new cases of chronic myeloid leukemia are diagnosed each year in the U.S.
Patients with chronic myeloid leukemia treated with TKIs can develop resistance or intolerance over time to these therapies.
Iclusig is a targeted cancer medicine designed to inhibit BCR-ABL, including drug-resistant mutants that arise during treatment.
Iclusig is the only TKI that has demonstrated activity against the T315I gatekeeper mutation of BCR-ABL, the most common mutation occurring in approximately 10% of patients with drug resistance.
The FDA approval of Iclusig was based on results from the pivotal phase 2 PACE ( Ponatinib Ph+ ALL and CML Evaluation ) trial in patients with chronic myeloid leukemia or Ph+ acute lymphoblastic leukemia who were resistant or intolerant to prior TKI therapy, or who had the T315I mutation of BCR-ABL.
Iclusig had robust anti-leukemic activity, with 54% of chronic-phase CML patients, including 70% of patients with the T315I mutation, achieving a major cytogenetic response ( MCyR ), the primary endpoint of the PACE trial for chronic-phase patients.
In patients with advanced disease, 52% of accelerated-phase CML patients, 31% of blast-phase CML patients and 41% of Ph+ acute lymphoblastic leukemia patients achieved a major hematologic response ( MaHR ) to Ponatinib.
MaHR was the primary endpoint in the trial for patients with advanced disease. ( Xagena )
Source: ARIAD, 2012
XagenaMedicine_2012
