FDA grants Exelixis XL184 orphan drug designation for treatment of medullary thyroid cancer

Exelixis, Inc. (Nasdaq:EXEL) today announced that the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to XL184 for treatment of follicular, medullary, and anaplastic thyroid carcinoma, and metastatic or locally advanced papillary thyroid cancer. A pivotal phase 3 trial of XL184 is ongoing in patients with medullary thyroid cancer (MTC) and the company expects to release top-line phase 3 results in the first-half of 2011 and potentially file a New Drug Application (NDA) for the compound in the second-half of 2011. Exelixis also is evaluating XL184 in phase 2 in solid tumors including metastatic castration-resistant prostate cancer, ovarian cancer, melanoma, breast cancer, non-small cell lung cancer, and hepatocellular cancer, and in a phase 1 trial in renal cell carcinoma and differentiated thyroid cancer. Additionally, a phase 2 trial in recurrent glioblastoma is ongoing.

“We are looking forward to advancing XL184 as a new potential treatment for MTC, for which we expect to file an NDA in the second half of 2011. This will be an important step toward advancing the care of patients with this cancer”

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 are looking forward to advancing XL184 as a new potential treatment for MTC, for which we expect to file an NDA in the second half of 2011. This will be an important step toward advancing the care of patients with this cancer," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "We intend to leverage the benefits associated with orphan drug status to support our aggressive development of XL184 in a broad number of cancers. We believe this will help position us towards achieving our goals of improving outcomes for patients in some of the largest cancer indications while realizing the commercial potential of this first-in-class dual MET/VEGFR inhibitor."

Cabozantinib Designated as Generic Name for XL184

Exelixis also announced that the United States Adopted Name Council (USANC) and the World Health Organization's INN programme have adopted cabozantinib as the generic name for XL184. The USANC is tri-sponsored by the American Medical Association (AMA), the United States Pharmacopeial Convention (USP), and the American Pharmacists Association (APhA), and establishes drug nomenclature classifications based on pharmacological and/or chemical relationships.