Even though Novartis A.G. discovered its Glivec tyrosine kinase inhibitor in 1992 before the proliferation of the current generation of discovery tools, its quick progress through the clinic illustrates the reason why good target validation will speed the drug development process. Glivec (STI571) entered the clinic to treat patients with chronic myelogenous leukemia in mid-1998, and Novartis plans to file for FDA approval early next year based on Phase II and ongoing Phase III results, bringing the total clinical development time to three years if approved on its first review.

Following strong Phase I efficacy data in patients with chronic stage CML, Novartis (SWX:NOVN; NVS, Basel, Switzerland) opted to run unusually large Phase II trials, up to 10 times larger than other companies running CML studies, in an expanded set of indications including accelerated and blast crisis stage patients. Next year's planned NDA submission is in part possible because of the large number of patients treated in Phase II.

The disease

CML is a malignancy of white blood cells (WBCs) in which WBC precursors take over the bone marrow, leading to leukocytosis, platelet disorders, anemia, splenomegaly, and eventually death.