The US Food and Drug Administration (FDA) has accepted ARIAD Pharmaceuticals' New Drug Application (NDA) filing for investigational BCR-ABL inhibitor, ponatinib, in patients with resistant or intolerant chronic myeloid leukaemia (CML) or Philadelphia-chromosome positive acute lymphoblastic leukaemia (Ph+ ALL). The FDA also has granted ARIAD’s request for Priority Review, which is given to investigational medicines that have the potential for providing significant improvement in the treatment, prevention, or diagnosis of a disease.

The FDA has established an action date of March 27, 2013 under the Prescription Drug User Fee Act (PDUFA).

Internally discovered at ARIAD, ponatinib is an investigational BCR-ABL inhibitor that also selectively inhibits certain other tyrosine kinases in preclinical studies, including FLT3, RET, KIT, and the members of the FGFR and PDGFR families of kinases.

The primary target for ponatinib is BCR-ABL, an abnormal tyrosine kinase that is expressed in chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). Ponatinib was designed using ARIAD’s computational and structure-based drug design platform to inhibit the activity of BCR-ABL with very high potency and broad specificity. Ponatinib targets not only native BCR-ABL but also its isoforms that carry mutations that confer resistance to treatment with existing tyrosine kinase inhibitors, including the T315I mutation for which no effective therapy currently exists.

CML is characterized by an excessive and unregulated production of white blood cells by the bone marrow due to a genetic abnormality that produces the BCR-ABL protein. After a chronic phase of production of too many white blood cells, CML typically evolves to the more aggressive phases referred to as accelerated phase or blast crisis. Ph+ ALL is a subtype of acute lymphoblastic leukemia that carries the Ph+ chromosome that produces BCR-ABL. It has a more aggressive course than CML and is often treated with a combination of chemotherapy and tyrosine kinase inhibitors. Because both of these diseases express the BCR-ABL protein, this would render them potentially susceptible to treatment with ponatinib.

ARIAD Pharmaceuticals, Inc. is focused on the discovery, development and commercialization of medicines to transform the lives of cancer patients.