ARIAD Completes Rolling Submission of New Drug Application for Ponatinib to the U.S. Food and Drug A
ARIAD Completes Rolling Submission of New Drug Application for Ponatinib to the U.S. Food and Drug Administration
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- ARIAD Pharmaceuticals, Inc. (NAS: ARIA) today announced it has completed the rolling submission of the New Drug Application (NDA) for its investigational BCR-ABL inhibitor, ponatinib, to the U.S. Food and Drug Administration (FDA). ARIAD provided the FDA with remaining chemistry, manufacturing, and controls (CMC) data. ARIAD is seeking U.S. marketing approval of ponatinib in patients with resistant or intolerant chronic myeloid leukemia (CML) and Philadelphia-chromosome positive acute lymphoblastic leukemia (Ph+ ALL). The Company has requested accelerated approval and a priority review of the ponatinib application by the FDA.
"In late July, we submitted the NDA for ponatinib ahead of schedule and, at the request of the FDA, in advance of having the final CMC data. We look forward to continuing our progress towards making ponatinib available to patients with CML and Ph+ ALL," stated Harvey J. Berger, M.D., chairman and chief executive officer of ARIAD. "If approved, we believe that ponatinib will become an important new medicine for CML and Ph+ ALL patients who have become resistant or intolerant to prior tyrosine kinase inhibitor therapy."
ARIAD anticipates approval and commercial launch of ponatinib in the U.S. in the first quarter of 2013. Also, the Marketing Authorization Application (MAA) for ponatinib, submitted in August, has been validated by the European Medicines Agency (EMA), commencing their review of the application. The Committee for Medicinal Products for Human Use (CHMP) has granted ARIAD's request for accelerated assessment of the MAA.
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.
About CML and Ph+ ALL
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 an emerging global oncology company focused on the discovery, development and commercialization of medicines to transform the lives of cancer patients. ARIAD's approach to structure-based drug design has led to several internally discovered, molecularly targeted product candidates for drug-resistant or difficult-to-treat cancers, including chronic myeloid leukemia and certain forms of non-small cell lung cancer. For additional information, visit http://www.ariad.com or follow ARIAD on Twitter (@ARIADPharm).
This press release contains "forward-looking statements" including, but not limited to, statements relating to the clinical data for ponatinib, submission, review, and potential approval of the NDA, and timing of other regulatory filings for marketing approvals. Forward-looking statements are based on management's expectations and are subject to certain factors, risks and uncertainties that may cause actual results, outcome of events, timing and performance to differ materially from those expressed or implied by such statements. These risks and uncertainties include, but are not limited to, preclinical data and early-stage clinical data that may not be replicated in later-stage clinical studies, the costs associated with our research, development, manufacturing and other activities, the conduct, timing and results of pre-clinical and clinical studies of our product candidates, the adequacy of our capital resources and the availability of additional funding, and other factors detailed in the Company's public filings with the U.S. Securities and Exchange Commission. The information contained in this press release is believed to be current as of the date of original issue. The Company does not intend to update any of the forward-looking statements after the date of this document to conform these statements to actual results or to changes in the Company's expectations, except as required by law.
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