A Targeted Therapy for a Rare Disease 

The FDA approved a kinase inhibitor for the treatment of inflammatory myofibroblastic tumors in children and adults. 

The U.S. Food and Drug Administration has approved crizotinib (Xalkori) for the treatment of certain patients 1 year of age or older with inflammatory myofibroblastic tumors (IMTs). The drug is intended for patients whose tumors have relapsed, are resistant to other therapy, or cannot be surgically removed, and that have the ALK protein on the cell surface. 

Crizotinib is a targeted therapy known as a kinase inhibitor. It blocks cancer-causing kinases such as ALK and ROS1, which can be mutated in several tumor types. Crizotinib was previously FDA-approved for the treatment of ALK-positive or ROS1-positive non-small cell lung cancer (NSCLC). 

The current approval was based on data from patients with IMTs selected from two multicenter, single-arm, open-label clinical trials—ADVL0912, which included 14 pediatric IMT patients, and A8081013, which included seven adult IMT patients. The objective response rate was 86 percent for the pediatric patients and 71.4 percent for the adult patients. 

IMTs are tumors that form in tissues that secrete mucus—like those in the eyes, nose, lungs, and reproductive tracts—or in the connective tissue between internal organs, called mesentery tissue. These tumors most commonly occur in children and young adults, but they can occur at any age. Although most IMTs are benign, they can sometimes invade or constrict nearby organs. Each year, around 150 to 200 people in the U.S. are diagnosed with IMTs. 

The FDA rendered its decision on July 14, 2022.