United States of America - The Food and Drug Administration (FDA) has granted approval of a new indication for temozolomide (Temodar®). The drug, used concurrently with radiotherapy and as maintenance therapy after radiotherapy, can extend the lives of adult patients newly diagnosed with glioblastoma multiforme (GBM), the most common form of malignant brain cancer.

GBM is usually fatal. The annual incidence of GBM is four to five cases per 100 000 persons with 8000 to 10 000 new cases diagnosed per year in North America.

The new approval of temozolomide for GBM was based on efficacy and safety data from a large randomized controlled study conducted by the European Organization for Research and Treatment of Cancer (EORTC) in patients with newly diagnosed GBM. Patients were randomized to treatment with radiation alone or to treatment with radiotherapy plus temozolomide. In the multi-centre trial of 573 patients, median survival was improved by two and a half months in the temozolomide group, a significant benefit. The median survival was 14.6 months with radiotherapy plus temozolomide and 12.1 months with radiotherapy alone.

Temozolomide was previously granted accelerated approval in 1999 for the treatment of adult patients with another form of brain tumour (anaplastic astrocytoma) in relapse after chemotherapy with nitrosurea and procarbazine.

Side effects for temozolomide reported include nausea, vomiting, headaches, fatigue, and anorexia. Preventive treatment for pneumocystis carinii pneumonia is required when temozolomide is administered with radiotherapy.

Reference: FDA Talk Paper, T05-07. 16 March 2005