The U.S. FDA approved Pfizer’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma that is anaplastic lymphoma kinase (ALK)-positive.
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The U.S. Food and Drug Administration (FDA) approved Pfizer’s Xalkori (crizotinib) for pediatric patients one year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is anaplastic lymphoma kinase (ALK)-positive. ALCL is a rare type of non-Hodgkin lymphoma that makes up about a third of cases of NHL in young people. About 90% of ALCL cases in young people are ALK-positive.
The approval was based on data from Study ADVL0912, a multicenter, single-arm, open-label trial in 121 patients ranging in ages from one to 21, and included 26 patients with r/r/, systemic ALK-positive ALCL after at least one systemic treatment. The data showed an objective response rate of 88%. In the 23 patients who achieved a response, 39% maintained that response for at least six months, and 22% maintained it for at least a year.
Xalkori is a tyrosine kinase inhibitor indicated since 2011 for patients with metastatic non-small cell lung cancer (NSCLC) whose tumors are ALK- or ROS1-positive. It has also been approved for that indication in more than 90 countries. It is also approved for ROS1-positive NSCLCL in more than 70 countries.
In the most recent quarter reported, Xalkori brought in $122 million, which is a drop of about 6% since the same period in 2019, when it brought in $130 million. It is facing sales pressure from Takeda’s Alunbrig (brigatinib) and Novartis’ Zykadia (ceritinib). For the full year of 2019, Xalkoria brought in $530 million, an increase of 1% from 2018.
“We are proud to deliver the first biomarker-driven therapy for children and young adults with ALCL,” said Chris Boshoff, chief development officer, Oncology, Pfizer Global Product Development. “Xalkori offers a meaningful new treatment option for young patients with relapsed or refractory ALK-positive ALCL. 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.”
The safety profile of the drug in this patient population was generally consistent with that in patients with ALK-positive and ROS1-positive metastatic NSCLC. The most common adverse reactions were diarrhea, vomiting, nausea, vision disorder, headache, musculoskeletal pain, stomatitis, fatigue, decreased appetite, pyrexia, abdominal pain, cough and pruritis.
Xalkoria received Breakthrough Therapy designation from the FDA in May 2018 for ALK-positive ALCL. The European Medicines Agency (EMA) has granted a Paediatric Investigational Plan (PIP) for the drug for pediatric patients with r/r systemic ALK-positive ALCL, which along with this approval, will pave the way for a potential regulatory submission in the European Union.
“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, associate professor of Pediatrics at the University of Pennsylvania/Children’s Hospital of Philadelphia and Principal Investigator of the pivotal study conducted via the Children’s Oncology Group. “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.”
