On July 14, 2022, the FDA approved the multikinase inhibitor Xalkori (crizotinib; from Pfizer) for the treatment of unresectable, recurrent, or refractory inflammatory myofibroblastic tumors with ALK mutation in children and adults.
Xalkori was previously approved for the treatment of patients with metastatic non–small-cell lung cancer and ALK or ROS1 mutation, and for young patients aged 1 to 21 years with relapsed or refractory systemic anaplastic large-cell lymphoma and ALK mutation.
This new indication was based on the results of 2 multicenter, single-arm, open-label clinical trials of patients with unresectable, recurrent, or refractory ALK-positive inflammatory myofibroblastic tumors.
One study included 14 young patients, and 12 of them had a response to treatment with Xalkori. The second study included 7 patients, 5 of whom had a response to treatment with Xalkori.
In the pediatric patients, the most common side effects were vomiting, nausea, diarrhea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edema, constipation, and headache. In the adult patients, the most common side effects were vision disorders, nausea, and edema.