Reason for request

New indication


Clinical Benefit


Considering all these elements, the Committee deems that the clinical benefit of XALKORI (crizotinib) hard capsules, in the 250 mg and 200 mg strengths, is substantial in the treatment of paediatric patients (age ≥6 to <18 years) with relapsed or refractory systemic anaplastic lymphoma kinase (ALK)-positive anaplastic large cell lymphoma (ALCL).

Clinical Added Value



  • the data from the phase 1-2 ADVL0912 and A8081013 studies suggesting an anti-tumour activity of crizotinib in paediatric patients with relapsed or refractory ALK-positive ALCL, with respective overall response rates of 88.5% (n=23/26) and 56.3% (n=9/16) and respective complete response rates of 80.8% (n=21/26) and 50.0% (n=8/16);
  • the partially met medical need in the second or later-line treatment of ALK-positive ALCL in children;
  • the expected, but not supported, improvement in care conditions compared to chemotherapy, with oral administration of crizotinib (hard capsules) at home;

and despite:

  • the absence of comparative data in the target paediatric population, in whom the results do not enable any robust conclusion to be reached with respect to the therapeutic contribution of crizotinib compared to the available alternatives;
  • the limited safety data in children in terms of the follow-up duration, with a median follow-up of less than 1 year in the studies provided;
  • the potential onset of ocular toxicity and grade 3-4 neutropenia, which are known adverse reactions of crizotinib occurring more frequently in children and requiring reinforced monitoring in the paediatric population;

the Committee deems that XALKORI (crizotinib) hard capsules, in the 250 mg and 200 mg strengths, provides a minor clinical added value (CAV IV) in the care pathway as second or later-line therapy for relapsed or refractory systemic ALK-positive anaplastic large cell lymphoma (ALCL) in paediatric patients (age ≥6 to <18 years).

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