FDA Approves Xalkori for Rare Type of Tumor

The FDA has approved Pfizer’s Xalkori (crizotinib) for an additional indication: to treat adult and pediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT). Myofibroblastic tumors, masses of immune cells, are rare.

The approval was based on safety and efficacy from two trials that included 14 pediatric patients from trial ADVL0912 and seven adult patients from trial A8081013. For the pediatric patients, 12 of the 14 patients experienced an objective response, For the adult patients, five patients had objective responses.

Among the pediatric patients, the most common adverse reactions were vomiting, nausea, diarrhea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edema, constipation, and headache. The most frequent adverse reactions in adult patients were vision disorders, nausea, and edema.

Xalkori, first approved in 2011, is also approved to treat ALK- or ROS1-positive non-small cell lung cancer and ALK-positive large cell lymphoma in children and young adults.