U.S. Food and Drug Administration
FDA Consumer magazine
July-August 2001
Table of Contents

FDA Approves Gleevec for Leukemia Treatment

The Food and Drug Administration has approved a promising new oral treatment for patients with chronic myeloid leukemia (CML)--a rare, life-threatening form of cancer--under its "accelerated approval" regulations. The FDA, which announced the approval on May 10, 2001, reviewed the marketing application for Gleevec (imatinib mesylate, also known as STI-571) in less than three months.

"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."

Accelerated approval allows the FDA to approve drugs for serious or life-threatening illnesses on the basis of clinical trials that indicate the drug is reasonably likely to be of real clinical benefit. Gleevec has been shown to reduce substantially the level of cancerous cells in the bone marrow and blood of treated patients.

The 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. The approval was based on three separate studies in about 1,000 patients. The FDA approved Gleevec under its 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 the FDA's acting commissioner, Bernard A. Schwetz, D.V.M, Ph.D. "However, 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. Side effects of Gleevec 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 2 percent of patients, and any unexpected and rapid weight gain should be investigated and, if necessary, treated.

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.

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.

Gleevec, a specific inhibitor of the translocation-created enzyme, works by blocking the rapid growth of white blood cells. The drug is manufactured by Novartis Pharma AG for Novartis Pharmaceuticals Corp., East Hanover, N.J.

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