United States of America - The Food and Drug Administration (FDA) has announced the approval of imatinib mesylate (Gleevec ®) tablets for the treatment of paediatric patients with Philadelphia chromosome positive (Ph+) chronic myeloid leukaemia (CML) in chronic phase - a rare, lifethreatening form of cancer that accounts for approximately two percent of all leukaemia in children.
Imatinib mesylate is indicated for children whose disease has recurred after stem-cell transplant or who are resistant to interferon alpha therapy. This drug was approved under the accelerated approval programme. As of yet, there are no controlled trials demonstrating clinical benefit, such as improvement in disease-related symptoms or increased survival. Subsequent studies after approval will be conducted to confirm that the drug has improved survival or other clinical benefits in paediatric patients.
In addition to its original approved indication for CML refractory to other treatments in adults, and expansion to use as a first line treatment for CML, imatinib mesylate was also previously granted accelerated approval for the treatment of gastrointestinal stromal cancer in February 2002. Accelerated approval for paediatric use is based on extrapolation of results from adults with CML together with good responses in a small number of children. As a condition of approval, the manufacturer has agreed to conduct paediatric studies after approval to gain greater insight into the drug’s use in children.
The most frequently reported adverse events reported with the use of imatinib mesylate are nausea, vomiting, diarrhoea, edema (sometimes severe), and muscle cramps. A considerable reduction in white blood cells and platelets was also reported.
Reference: FDA News, P03 - 43 (2003).