FDA approves new treatment for rare tumour

pharmafile | July 15, 2022 | News story | Medical Communications  

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

This marks the fourth approval of the tyrosine kinase inhibitor from Pfizer by the FDA. The safety and efficacy of crizotinib were evaluated in two multicentre, single-arm, open-label trials that included 14 paediatric patients from the clinical trial ADVL0912 (NCT00939770), and seven adult patients from the trila A8081013 (NCT01121588) with unresectable, recurrent, or refractory ALK-positive IMT.

IMT appears in organs such as the lungs, stomach, bladder, or liver. These tumours are not cancerous, and do not usually spread. However, local growth within organs can cause problems for patients.

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

Meanwhile, the most frequent advere reactions in adult patients were vision disorders, nausea, and edema. Edema is the medical term for swelling caused by excess fluid trapped in the body’s tissues.

The major efficacy outcome measure of these trials was objective response rate (ORR). Among the paediatric patients, a total of 12 of the 14 patients (86%) experienced an objective response, assessed by an independent review committee. For the seven adult patients, five had objective responses.

The drug is already approved to treat metastatic non-small cell lung cancer (NSCLC) in patients whose tumours are positive for ALK or ROS1 mutations, alongside for ALK-positive anaplastic large cell lymphoma in children and young adults. Crizotinib, marketed as Xalkori, was first introduced over a decade ago. The recommended dose for adults in 250mg orally twice daily, until disease progression or unacceptable toxicity.

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