The U.S. Food and Drug Administration (FDA) Wednesday expanded the approval of Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after the failure of other therapies.

cGVHD is a life-threatening condition that occurs when cells from a stem cell transplant attack healthy cells in a patient’s tissues. Symptoms of cGVHD can be found in the skin, mouth, eyes, gut, liver, and lungs, and is estimated to occur in 30-70 percent of all patients who receive hematopoietic stem cell transplantation (HSCT).

The approval of Imbruvica for treating cGVHD was studied in a single-arm trial of 42 patients with cGVHD whose symptoms kept persisting despite taking standard treatment with corticosteroids. Most patients suffered mouth ulcers, and skin rashes, with 50 percent of patients had two or more organs affected. In the trial, 67 percent of patients experienced improvements. In 48 percent of patients in the trial, the improvement lasted for up to five months or longer.

This is the first FDA-approved therapy for the treatment of cGVHD.

Common side effects of Imbruvica include bruising, diarrhea, fatigue, low levels of blood platelets (thrombocytopenia), muscle spasms, swelling and sores in the mouth (stomatitis), nausea, severe bleeding (hemorrhage), low levels of red blood cells (anemia) and lung infection (pneumonia).

Serious side effects of Imbruvica include infections, severe bleeding, low levels of blood cells, high blood pressure, irregular heartbeat, new cancers (second primary malignancies) and metabolic abnormalities. Women who are pregnant or breastfeeding should not take Imbruvica because it may cause harm to a developing fetus or a newborn baby.

“Patients with cGVHD who do not respond to other forms of therapy --typically corticosteroids to suppress their immune system -- now have a treatment option specifically indicated to treat their condition,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence. “This approval highlights how a known treatment for cancer is finding a new use in treating a serious and life-threatening condition that may occur in patients with blood cancer who receive a stem cell transplant.”

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