FDA Approves Crizotinib For ALK-Positive Anaplastic Large Cell Lymphoma in Children


Crizotinib is the first biomarker-driven therapy for relapsed or refractory anaplastic large cell lymphoma in children and young adults.

The FDA has approved crizotinib (Xalkori, Pfizer) as the first biomarker-driven therapy for relapsed or refractory anaplastic large cell lymphoma (ALCL) in children and young adults. The treatment is indicated for pediatric patients 1 year of age and older with relapsed or refractory ALCL that is anaplastic lymphoma kinase (ALK)-positive.

According to a press release, ALCL is a rare form of non-Hodgkin lymphoma (NHL) and accounts for approximately 30% of NHL cases in young people. Approximately 90% of ALCL cases in pediatric patients and young adults are ALK-positive.

The FDA approval is based on results from Study ADVL0912, a multicenter, single arm, open-label study of 121 patients between 1 and 21 years of age, including 26 patients with relapsed or refractory, systemic ALK-positive ALCL after at least 1 systemic treatment. Treatment with crizotinib resulted in an objective response rate of 88%, and among the 23 patients who achieved a response, 39% maintained their response for at least 6 months and 22% maintained their response for at least 12 months.

“We are proud to deliver the first biomarker-driven therapy for children and young adults with ALCL,” said Christ Boshoff, MD, PhD, chief development officer of oncology at Pfizer Global Product Development, in a press release. “Xalkori offers a meaningful new treatment option for young patients with relapsed or refractory ALK-positive ALCL.”

The press release noted that although the majority of people with ALK-positive ALCL respond well to chemotherapy and experience long-term remission, many patients will unfortunately relapse or require alternative treatment approaches.

“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 Yael Mosse, MD, principal investigator for the pivotal study run through the University of Pennsylvania/Children’s Hospital of Philadelphia Children’s Oncology Group, in a press 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.”

The safety profile of crizotinib in ALK-positive ALCL in children and young adults is generally consistent with the profile observed in patients with ALK-positive and ROS1-positive metastatic non-small cell lung cancer. The most common adverse reactions included diarrhea, vomiting, nausea, vision disorder, headache, musculoskeletal pain, stomatitis, fatigue, and decreased appetite. The most common grade 3 or 4 laboratory abnormalities included neutropenia, lymphopenia, and thrombocytopenia.

“Crizotinib represents an exciting new development in the treatment of this disease,” said Meghan Gutierrez, chief executive officer at the Lymphoma Research Foundation, 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. Today’s news builds upon this progress and provides hope to pediatric patients with ALCL and their loved ones.”


Pfizer’s Xalkori (Crizotinib) Approved By FDA For ALK-Positive Anaplastic Large Cell Lymphoma in Children and Young Adults [news release]. Pfizer; January 14, 2021. https://investors.pfizer.com/investor-news/press-release-details/2021/Pfizers-XALKORI-crizotinib-Approved-by-FDA-for-ALK-positive-Anaplastic-Large-Cell-Lymphoma-in-Children-and-Young-Adults/default.aspx. Accessed January 15, 2021.

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