FDA APPROVES GLEEVEC FOR LEUKEMIA TREATMENT

The Food and Drug Administration today announced the approval of Gleevec (imatinib mesylate, also known as STI-571), a promising new oral treatment for patients with chronic myeloid leukemia (CML) -- a rare life-threatening form of cancer. FDA reviewed the marketing application for Gleevec in less than three months under its "accelerated approval" regulations.

"FDA and Novartis, the drug's manufacturer, should be commended for the rapid development and review that will make this product available soon for the leukemia patients who desperately need it," said Health and Human Services Secretary Tommy G. Thompson. "It is also important to recognize that today's approval is also a culmination of years of work and years of investment, by many people in many different institutions, and even in different fields of medicine. It's a testament to the groundbreaking scientific research taking place in labs throughout America."

FDA approved the drug for treating patients with three stages of CML: CML myeloid blast crisis, CML accelerated phase, or CML in chronic phase after failure of interferon treatment. Accelerated approval allows FDA to approve drugs for serious or life-threatening illnesses on the basis of clinical trials establishing that the drug has an effect on a surrogate endpoint that is reasonably likely to predict a real clinical benefit. Gleevec has been shown to reduce substantially the level of cancerous cells in the bone marrow and blood of treated patients.

FDA approved Gleevec under FDA’s orphan drug program which provides financial incentives for drugs developed to treat rare diseases.

“Although the long term benefits of the drug are not yet known, early studies have shown that Gleevec will offer a significant improvement for many patients,” said FDA’s acting commissioner, Bernard A. Schwetz, D.V.M., Ph.D.

“However,” he said, “further studies are needed to evaluate whether Gleevec provides an actual clinical benefit, such as improved survival, as well as to examine its effect when used in early stage disease.”

Dr. Schwetz also said that it is important for physicians and patients to understand currently known side effects from Gleevec, and to realize that additional side effects may be discovered with more follow-up of patients in ongoing studies.

Chronic myeloid leukemia occurs when pieces of 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.

“For the first time, cancer researchers now have the necessary tools to probe the molecular anatomy of tumor cells in search of cancer-causing proteins,” said Richard Klausner, M.D., director of the National Cancer Institute. “Gleevec offers proof that molecular targeting works in treating cancer, provided that the target is correctly chosen. The challenge now is we’ve got to find these targets.”

Symptoms of leukemia may include abdominal discomfort, bone and joint pains, and fatigue. Some patients are diagnosed when a routine blood test reveals a high white blood cell count with increased numbers of immature white blood cells.

Today’s approval of Gleevec for treating the three phases of CML was based on three separate single arm studies in about 1000 patients.

These clinical trials were not designed to determine whether Gleevec improves survival. The sponsor is currently accruing patients for follow-up studies necessary to confirm clinical effectiveness of Gleevec.

Side effects frequently reported in trials include nausea, vomiting, edema (fluid retention), muscle cramps, skin rash, diarrhea, heartburn, and headache. Severe fluid retention occurred in up to two percent of patients, and any unexpected and rapid weight gain should be investigated and, if necessary, treated.

The drug is manufactured by Novartis Pharma AG for Novartis Pharmaceuticals Corporation, East Hanover, New Jersey. It is expected to be available by the end of May, 2001.

For more information on this subject, see the Gleevec (Imatinib Mesylate) Drug Information Page on FDA's Center for Drug Evaluation and Research Website.

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