The Food and Drug Administration (FDA) has approved Xalkori (crizotinib) for the treatment of patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory myofibroblastic tumor (IMT) that is anaplastic lymphoma kinase (ALK)-positive.
The approval was based on data from 2 multicenter, single-arm, open-label trials, ADVL0912 (ClinicalTrials.gov Identifier: NCT00939770) and A8081013 (ClinicalTrials.gov Identifier: NCT01121588), which included 14 pediatric and 7 adult patients, respectively, with unresectable, recurrent or refractory ALK-positive IMT. Pediatric patients received crizotinib 280mg/m2 twice daily, and adults received 250mg twice daily. The major efficacy outcome for both studies was objective response rate, as assessed by an independent review committee.
Results showed that among the 14 pediatric patients, 12 (86%; 95% CI, 57-98) achieved an objective response, including 5 with a complete response and 7 with a partial response. Among the 12 patients who experienced a response, 7 maintained the response for at least 6 months and 7 maintained the response for at least 12 months.
For the 7 adult patients, 5 patients achieved an objective response, including 1 with a complete response. The duration of response was at least 6 months for all 5 patients and 2 patients maintained the response for at least 12 months.
The most common adverse reactions reported in pediatric patients were vomiting, nausea, diarrhea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edema, constipation, and headache. The most common adverse reactions observed in adult patients were vision disorders, nausea, and edema.
Xalkori, a tyrosine kinase inhibitor, is also indicated for the treatment of patients with metastatic non-small cell lung cancer that is ALK or ROS1-positive, and for the treatment of relapsed or refractory systemic anaplastic large cell lymphoma that is ALK-positive.
- FDA approves crizotinib for ALK-positive inflammatory myofibroblastic tumor. News release. US Food and Drug Administration. Accessed July 14, 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-crizotinib-alk-positive-inflammatory-myofibroblastic-tumor
- Xalkori. Package insert. Pfizer Inc.; 2022. Accessed July 14, 2022. https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/202570s033lbl.pdf