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12:00 AM
Dec 14, 2009
 |  BioCentury  |  Product Development

Covering all the bases

Detailed Phase III data presented by Novartis AG at the American Society of Hematology meeting last week will provide the first test of whether regulators will accept major molecular response as a new endpoint for trials of compounds to treat chronic myelogenous leukemia.

The data will be used to support applications for the company's Tasigna nilotinib as first-line treatment for newly diagnosed Philadelphia chromosome-positive CML. Novartis plans to submit an sNDA to FDA and a Type II variation application to EMEA by month's end.

Major molecular response (MMR) is a more quantitative endpoint that can be measured with a less invasive procedure than cytogenetic response, the current standard.

Early chemotherapies for CML, such as the generic busulfan, were approved based on hematologic response as measured by a blood panel to count white blood cells.

More recent targeted therapies such as tyrosine kinase inhibitors, including Novartis' Gleevec imatinib and Tasigna, were approved based on the surrogate endpoint of cytogenetic response (CCyR).

Gleevec, a BCR-ABL tyrosine kinase inhibitor, is approved for adults with newly diagnosed Ph+ CML in chronic phase, Ph+ CML in accelerated or blast crisis phase, or patients in chronic phase after failure of interferon alpha therapy. Gleevec also is approved for use in pediatric patients with newly diagnosed Ph + CML.

Tasigna, a second-generation BCR-ABL tyrosine kinase inhibitor, is approved for Ph+ CML with resistance or intolerance to prior therapy, including Gleevec.

CCyR requires taking a bone marrow biopsy and then visually counting cells containing the Ph chromosome under a...

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