FDA Approves Xalkori for Children, Young Adults With Relapsed or Refractory Anaplastic Large Cell Lymphoma

The Food and Drug Administration (FDA) on Thursday approved the supplemental new drug application for Xalkori (crizotinib) to treat children aged 1 year or older and young adults with relapsed or refractory, systemic ALK-positive anaplastic large cell lymphoma (ALCL), according to the agent’s manufacturer, Pfizer.

“We are proud to deliver the first biomarker-driven therapy for children and young adults with ALCL. Xalkori offers a meaningful new treatment option for young patients with relapsed or refractory ALK-positive ALCL,” said Dr. Chris Boshoff, chief development officer of oncology at Pfizer Global Product Development, said in a company-issued press release. “Xalkori transformed the treatment of ALK-positive non-small cell lung cancer as the first biomarker-driven therapy for that disease, and this approval is a notable milestone in our journey to continue to follow the science to address cancers with significant unmet need.”

A rare form of non-Hodgkin lymphoma (NHL), ALCL makes up approximately 30% of cases of NHL in the younger patient population, according to the release.

The agency’s decision was based on data from a multicenter, single-arm, open-label study.

In the ADVL0912 trial, 26 patients between the ages of 1 and 21 with relapsed or refractory, systemic ALK-positive ALCL were enrolled, all of whom had at least one prior systemic therapy. In these participants, treatment with Xalkori induced an objective response rate (the proportion of patients who had a complete or partial response to treatment) of 88%.

Among those who achieved a response, 39% maintained their response to therapy for at least six months and 22% maintained response for at least one year.

The safety profile of the drug in this patient population was consistent with what has been observed in patients with ALK-positive and ROS-1 positive metastatic non-small cell lung cancer. The most common side effects, which occurred in more than 35% of patients, included, but were not limited to: diarrhea, vomiting, nausea, headache, fatigue, decreased appetite, abdominal pain and cough.

“With increased attention being placed on the development of targeted agents and the importance of ALK in pediatric patients with ALCL, the approval of Xalkori is a significant victory in our ongoing fight against these cancers that provides an outpatient oral medication with the real possibility of robust and sustained responses,” said the study’s principal investigator Dr. Yael Mossé, an associate professor of pediatrics at the University of Pennsylvania and Children’s Hospital of Philadelphia, in the release. “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 is currently approved for the treatment of patients aged 1 year and older and young adults with relapsed or refractory, systemic ALK-positive ALCL. The agent’s manufacturer notes that there is no safety and efficacy data in adults with the disease.

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