GLEEVEC APPROVED FOR FIRST LINE TREATMENT OF CHRONIC MYELOID LEUKEMIA (CML)

The Food and Drug Administration (FDA) today announced the approval of Gleevec (imatinib mesylate) for the first-line treatment of patients with chronic myeloid leukemia (CML), an uncommon life-threatening form of cancer-- affecting about 40,000 people in the United States.

Gleevec was first approved in May 2001 for the advanced stages of CML under FDA's accelerated approval regulations. The drug was also indicated for use as a second-line treatment for chronic phase patients after failure of interferon-alpha therapy. At that time, further studies were needed to evaluate whether the drug provided an actual clinical benefit, such as improved survival, as well as to examine its effect when used in early stage disease.

"Today's approval represents continued efforts by government and industry to provide patients suffering from CML with additional therapies that have proven safe and effective through on-going research and clinical trials", said FDA Commissioner Mark B. McClellan, M.D., Ph.D. "With this new use even more patients will have access to this product earlier on in their fight against cancer," he said.

Chronic myeloid leukemia occurs when two different chromosomes break off and reattach on the opposite chromosome, forming the so called "Philadelphia chromosome". This chromosome translocation leads to a blood cell enzyme being "turned on" all the time. As a result, potentially life-threatening levels of both mature and immature white blood cells occur in the bone marrow and the blood.

Gleevec, a specific inhibitor of the translocation-created enzyme, works by blocking the rapid growth of white blood cells. Approval was based on a clinical trial of 1106 patients with newly diagnosed CML (chronic phase). Five hundred and fifty three patients were treated with Gleevec and 553 were given standard CML therapy with a combination of interferon-alpha and cytarabine. The patients treated with Gleevec after one year had significantly fewer cancerous cells in their blood and bone marrow. The rate of progression of disease was also decreased in the patients treated with Gleevec. Because patients with CML often live for up to 10 years with the disease, the 14-month median length of follow-up was too short to measure long term clinical benefits such as improved survival.

Gleevec is now approved for the treatment of patients with all three stages of CML -- CML myeloid blast crisis, CML accelerated phase, and CML in chronic phase, either before or after use of other therapy. The only known cure for CML is still by a stem cell (bone marrow) transplant.

The most common side effects reported with use of Gleevec include nausea, vomiting, edema (fluid retention), muscle cramps, fatigue, skin rash, and headache.

Gleevec is designated as an orphan drug. Orphan drugs are developed to treat rare diseases, that is, conditions that affect fewer than 200,000 people in the U.S. The Orphan Drug Act provides a seven-year period of exclusive marketing for the drug for the orphan use to the first sponsor who obtains marketing approval for a designated orphan drug.

Gleevec is also approved for the treatment of gastrointestinal stromal cancer and is manufactured by Novartis Pharma AG for Novartis Pharmaceuticals Corporation located in East Hanover, N.J.

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