The US Food and Drug Administration (FDA) has approved Pfizer’s supplemental New Drug Application (sNDA) for Xalkori (crizotinib) to treat a type of lymphoma.

The FDA approval indicated Xalkori for the treatment of anaplastic lymphoma kinase (ALK)-positive relapsed systemic anaplastic large cell lymphoma (ALCL) in paediatric patients and young adults, aged one year and above.

Xalkori is a tyrosine kinase inhibitor (TKI), used in treating ALK- or ROS1-positive metastatic NSCLC tumours, as detected by an FDA-approved test.

The drug has been approved for ALK-positive NSCLC in more than 90 countries including the US, Australia, Canada, China, Japan, South Korea and the EU, and for ROS1-positive NSCLC in more than 70 countries.

In May 2018, the FDA has granted Breakthrough Therapy designation (BTD) Xalkori for the ALK-positive ALCL indication, and the European Medicines Agency (EMA) has agreed to prepare a Paediatric Investigational Plan (PIP) for the drug.

Pfizer global product development oncology chief development officer Chris Boshoff said: “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.

“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.”

ALCL is a rare form of non-Hodgkin lymphoma (NHL) that accounts for around 30% of NHL cases in young people, while nearly 90% of ALCL cases in young people are ALK-positive.

The US FDA approved Xalkori based on results from ADVL0912 study, a multicentre, single-arm, open-label study in 121 patients, aged between 1 and 21, with relapsed or refractory, systemic ALK-positive ALCL.

In the study, treatment using Xalkori resulted in an objective response rate of 88% and showed a consistent safety profile with that observed in patients with ALK-positive and ROS1-positive metastatic NSCLC.

Except for laboratory abnormalities, the most common adverse reactions include diarrhoea, vomiting, nausea, vision disorder, headache, musculoskeletal pain, stomatitis, fatigue, decreased appetite, pyrexia, abdominal pain, cough and pruritis.

Children’s Oncology Group study principal investigator Yael Mossé said: “With increased attention being placed on the development of targeted agents and the importance of ALK in paediatric 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.

“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.”