Exelixis' Cabozantinib Granted Orphan Drug Designation for the Treatment of Hepatocellular Carcinoma
Tuesday, March 07, 2017
Exelixis, Inc. announced that the U.S. Food & Drug Administration (FDA) has granted orphan drug designation to cabozantinib for the treatment of hepatocellular carcinoma (HCC). This information was posted to FDA’s website on March 4, 2017 and can be accessed here. A pivotal phase 3 trial (CELESTIAL) of cabozantinib is ongoing in patients with advanced HCC, and Exelixis has guided that data from the trial are expected in 2017.
Orphan drug status is granted to treatments for diseases that affect fewer than 200,000 people in the U.S. and provides certain incentives for medications intended for the treatment, diagnosis or prevention of rare diseases. At present, these incentives include seven years of marketing exclusivity for the orphan indication, certain federal grants, tax credits and waiver of certain FDA fees.
