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FDA Approves Xalkori (crizotinib) for ALK-Positive Inflammatory Myofibroblastic Tumors

On July 14, 2022, the Food and Drug Administration approved crizotinib (Xalkori, Pfizer Inc.) for adult and pediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory anaplastic lymphoma kinase (ALK)-positive myofibroblastic tumors (IMT).

The safety and efficacy of crizotinib were evaluated in two multicenter, single-arm, open-label trials that included 14 pediatric 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 pediatric 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 pediatric patients were vomiting, nausea, diarrhea, abdominal pain, rash, vision disorder, upper respiratory tract infection, cough, pyrexia, musculoskeletal pain, fatigue, edema, constipation, and headache. The most frequent adverse reactions (≥35%) in adult patients were vision disorders, nausea, and edema.

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

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.

This application was granted priority review. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. Crizotinib received orphan drug designation.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.

For information on the COVID-19 pandemic, see the following resources:

Source: FDA

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