Xalkori received Breakthrough Therapy designation for the ALK-positive ALCL indication in May 2018 and if approved, would be the first biomarker-driven therapy for this type of pediatric lymphoma.
The Prescription Drug User Fee Act (PDUFA) goal date for a decision by the FDA is January 2020.
Anaplastic large cell lymphoma is a rare type of non-Hodgkin lymphoma, divided into ALK-positive or ALK-negative disease.
Though the five-year survival rate for children with cancer in the US is now the highest it's ever been at 80%, children with cancer continue to face challenges in treating their disease, including rare tumor types, variations in medicine response and prolonged risk of side effects.
The FDA submission is supported by the results from Study ADVL0912 (NCT00939770) and Study A8081013 (NCT01121588).
Study ADVL0912 is a Phase 1/2 study conducted in collaboration with the Children's Oncology Group, evaluating the maximum dose that is safe and tolerable, and assessing clinical activity in pediatric patients with relapsed or refractory solid tumors and ALCL.
Pfizer provided funding and support to COG for this trial.
Study A8081013 evaluated Xalkori in pediatric and adult patients with advanced malignancies known to be ALK-positive other than non-small cell lung cancer (NSCLC) and included patients with relapsed or refractory ALCL.
These two studies showed compelling antitumor activity in pediatric and adult patients who received Xalkori.
This FDA submission is in addition to the European Medicines Agency's agreement on a Pediatric Investigational Plan for Xalkori including the treatment of pediatric patients with relapsed or refractory systemic ALK-positive ALCL.
This decision represents a step forward for potential regulatory submission for Xalkori in pediatric patients with relapsed or refractory ALK-positive ALCL in the European Union.
Xalkori is a tyrosine kinase inhibitor indicated for the treatment of patients with metastatic NSCLC whose tumors are ALK-positive or ROS1-positive as detected by an FDA-approved test.
Xalkori has received approval for patients with ALK-positive NSCLC in more than 90 countries worldwide including Australia, Canada, China, Japan, South Korea and the European Union.
Xalkori is also approved for ROS1-positive NSCLC in more than 70 countries.
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