FDA has granted Orphan Drug Designation to XALKORI (crizotinib) for the treatment of ROS1-positive non-small cell lung cancer (NSCLC).  XALKORI is a small-molecule tyrosine kinase inhibitor of ALK, ROS1, and another proto-oncogene receptor tyrosine kinase, MET.  It is manufactured by Pfizer, a global pharmaceutical giant and is currently approved in the US for the treatment of patients with metastatic NSCLC whose tumors are ALK-positive.  This designation follows the Breakthrough Therapy Designation in April this year for the same indication.  ROS1-positive NSCLC represents a particular molecular subgroup of NSCLC and clinical studies have shown that XALKORI exhibited antitumor activities in this group of patients.  NSCLC is a heterogeneous disease and the coupling of XALKORI treatment with companion diagnostic tests further approved the importance of precision medicine.



  1. http://www.pfizer.com/news/press-release/press-release-detail/pfizer_receives_u_s_fda_breakthrough_therapy_designation_for_xalkori_crizotinib_for_the_treatment_of_patients_with_ros1_positive_non_small_cell_lung_cancer
  2. http://pubs.acs.org/doi/abs/10.1021/jm500261q
  3. http://www.pfizer.com/products/product-detail/xalkori