The Food and Drug Administration has approved crizotinib for adult and paediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumours (IMT).

The safety and efficacy of crizotinib were evaluated in two multicentre, single-arm, open-label trials that included 14 paediatric patients from trial ADVL0912 (NCT00939770) and 7 adult patients from trial A8081013 (NCT01121588) with unresectable, recurrent, or refractory ALK-positive IMT.

The major efficacy outcome measure of these trials was objective response rate (ORR). For the 14 paediatric patients, a total of 12 of the 14 patients (86%, 95% CI: 57, 98) experienced an objective response, assessed by an independent review committee. For the 7 adult patients, 5 patients had objective responses.

The most common adverse reactions (≥35%) in paediatric patients were vomiting, nausea, diarrhoea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edoema, constipation, and headache.

The most frequent adverse reactions (≥35%) in adult patients were vision disorders, nausea, and edoema.

The recommended crizotinib dose in adult patients is 250 mg orally twice daily until disease progression or unacceptable toxicity. The recommended paediatric dose is 280 mg/m2 orally twice daily until disease progression or unacceptable toxicity.

View full prescribing information for crizotinib.

Source: FDA