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FDA Grants Orphan Drug Designation to Anticancer Agent XL119

SOUTH SAN FRANCISCO, Calif., March 15 /PRNewswire-FirstCall/ -- Exelixis, Inc. announced that the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to XL119, an experimental treatment for bile duct tumors. The company anticipates initiating a pivotal Phase 3 clinical trial for XL119 in this indication in the second quarter of 2004. Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the U.S. and provides the benefits of extended market exclusivity for seven years, tax credits of up to 50% of the qualified clinical trial expenses and a waiver of FDA user fees.

"We have made considerable progress toward initiating the Phase 3 trial of XL119 as a potential treatment for bile duct cancer and anticipate beginning to open trial centers and enroll subjects within the next few months," said George A. Scangos, Ph.D., president and chief executive officer. "For the 30,000 people worldwide who develop bile duct cancer each year and for whom there are currently no approved therapies, we are hopeful that XL119 will prove beneficial. We are appreciative of the support provided by the FDA for developing potential treatments in orphan indications."

In September of 2003, Exelixis reached an agreement with the FDA through the Special Protocol Assessment (SPA) process on the design of the XL119 Phase 3 clinical trial. Based on the SPA, the trial will be a randomized, well-controlled, comparative study of XL119 in several hundred patients as a single-agent therapy with a survival-based endpoint. The company also intends to continue to work with the National Cancer Institute in planning additional Phase 2 exploratory studies of XL119 in various tumor types where the compound may demonstrate potential benefit. The company may also consider applying for European orphan drug designation for XL119 following the commencement of the Phase 3 clinical trial in Europe.

Source: Exelixis, Inc.

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