Favourable opinion for reimbursement of XALKORI (crizotinib) in the treatment of paediatric patients (age ≥6 to <18 years) with recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT).
Role in the care pathway?
In paediatric medicine, despite the low level of evidence related to the limited data and the resulting uncertainties with respect to its efficacy, the Committee considers that XALKORI (crizotinib) is a treatment option for recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumours (IMT) in children from 6 years of age.
According to the experts, XALKORI (crizotinib) could have a role in the paediatric management of:
ALK-positive IMT that cannot be operated on using conservative surgery following the failure of anti-inflammatory drugs;
metastatic ALK-positive IMT.
Clinical Benefit
Substantial
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 recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT).
Clinical Added Value
minor
Considering:
the data from phase 1-2 studies provided suggesting an anti-tumour activity of crizotinib in recurrent or refractory ALK-positive unresectable IMTs, in particular the results of the ADVL0912 study reporting the following in 12 paediatric patients treated at a dose of 280 mg/m² twice daily (MA dosage): 5 patients with a complete response, 6 patients with a partial response and 1 patient with stable disease, and a median response duration of 14.8 months (min./max.: 8-48.9 months);
the inadequately met medical need in the treatment of recurrent or refractory ALK-positive unresectable IMTs in children due to the absence of a standard and/or authorised treatment in the event of IMT that cannot be operated on using conservative surgery or metastatic IMT;
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 for recurrent or refractory anaplastic lymphoma kinase (ALK)-positive unresectable inflammatory myofibroblastic tumour (IMT) in paediatric patients (age ≥6 to <18 years).
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