The Food and Drug Administration (FDA) has approved Xalkori® (crizotinib; Pfizer) for the treatment of pediatric patients aged 1 year and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. The safety and efficacy of the treatment has not been established in older adults with ALK-positive ALCL.
The approval was based on data from an open-label phase 1/2 trial in patients aged 1 to 21 years that included 26 patients with relapsed or refractory, systemic ALK-positive ALCL after at least 1 systemic treatment. Patients received crizotinib 280mg/m2 (n=20) or 165mg/m2 (n=6) orally twice daily until disease progression or unacceptable toxicity. Patients were permitted to discontinue crizotinib to undergo hematopoietic stem cell transplantation.
Results showed an objective response rate of 88% (95% CI, 71-96) with crizotinib; complete remission was observed in 81% of patients (n=21). Among the 23 patients who achieved a response, 39% (n=9) maintained the response for at least 6 months and 22% (n=5) maintained the response for at least 12 months.
As for safety, the most common adverse reactions (incidence of greater than or equal to 35%), excluding laboratory abnormalities, were diarrhea, vomiting, nausea, vision disorder, headache, musculoskeletal pain, stomatitis, fatigue, decreased appetite, pyrexia, abdominal pain, cough, and pruritus. The most common grade 3 or 4 laboratory abnormalities (incidence of greater than or equal to 15%) were neutropenia, lymphopenia, and thrombocytopenia.
Commenting on the approval, Yael Mossé, MD, Associate Professor of Pediatrics at the University of Pennsylvania/ Children’s Hospital of Philadelphia and Principal Investigator for the pivotal study run through the Children’s Oncology Group, said: “ALK fusions play an important role in the pathology of ALCL, and it’s exciting that Xalkori is able to leverage this dependence to provide a treatment option for young people faced with ALCL disease progression.”
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 as detected by an FDA-approved test.
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1. Pfizer’s Xalkori® (crizotinib) approved by FDA for ALK-positive anaplastic large cell lymphoma in children and young adults. [press release]. New York, NY: Pfizer, Inc.; January 14, 2021.
2. FDA approves crizotinib for children and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma. [press release]. Silver Springs, MD: US Food and Drug Administration; January 15, 2021.
3. Xalkori [package insert]. New York, NY: Pfizer, Inc.; 2021.