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Xalkori Approved for Pediatric Patients and Young Adults with Relapsed or Refractory ALK-Positive ALCL

On January 14, 2021, the FDA approved crizotinib (Xalkori; Pfizer), an oral tyrosine kinase inhibitor, for the treatment of pediatric patients aged ≥1 years and young adults with relapsed or refractory systemic ALK-positive anaplastic large-cell lymphoma (ALCL). The safety and efficacy of crizotinib have not been established in older adults with relapsed or refractory systemic ALK-positive ALCL.

“Crizotinib represents an exciting new development in the treatment of this disease,” said Meghan Gutierrez, Chief Executive Officer, Lymphoma Research Foundation, New York City, in a press release. “Researchers have made significant progress in our understanding of ALCL, which we hope will continue to improve treatment strategies and the options for children with ALCL.”

The approval of crizotinib was based on results of a multicenter, single-arm, open-label phase 1/2 trial of 121 patients aged 1 to ≤21 years that included 26 patients with relapsed or refractory systemic ALK-positive ALCL after ≥1 systemic treatments.

Patients received crizotinib dosed at 280 mg/m2 (N = 20) or 165 mg/m2 (N = 6) orally twice daily until disease progression or unacceptable toxicity. Patients could discontinue crizotinib to undergo hematopoietic stem-cell transplantation.

The ORR in the 26 patients was 88%, with a complete remission rate of 81%. Of the 23 patients who achieved a response, 39% maintained their response for ≥6 months and 22% maintained their response for ≥12 months.

The most common (≥35%) adverse reactions, 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 (≥15%) grade 3/4 laboratory abnormalities were neutropenia (62%), lymphopenia (35%), and thrombocytopenia (19%).

The FDA previously granted crizotinib a priority review for this indication.

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