The U.S. Food and Drug Administration expanded the approval of Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft versus host disease (cGVHD) after failure of one or more treatments. This is the first FDA–approved therapy for the treatment of cGVHD.

“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, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “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.”

The efficacy and safety of Imbruvica for the treatment of cGVHD were studied in a single–arm trial of 42 patients with cGVHD whose symptoms persisted despite standard treatment with corticosteroids. Most patients’ symptoms included mouth ulcers and skin rashes, and more than 50 percent of patients had two or more organs affected by cGVHD. In the trial, 67 percent of patients experienced improvements in their cGVHD symptoms. In 48 percent of patients in the trial, the improvement of symptoms lasted for up to five months or longer.

Common side effects of Imbruvica in patients with cGVHD include fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, swelling and stomatitis, nausea, hemorrhage, anemia and pneumonia.

Serious side effects of Imbruvica include severe bleeding, cytopenias, atrial fibrillation, hypertension, second primary malignancies and tumor lysis syndrome. Women who are pregnant or breastfeeding should not take Imbruvica because it may cause harm to a developing fetus or a newborn baby.

Imbruvica, a kinase inhibitor, was previously approved for certain indications in treating chronic lymphocytic leukemia, Waldenström’s macroglobulinemia and marginal zone lymphoma, as well as under accelerated approval status for mantle cell lymphoma.

The FDA granted this application Priority Review and Breakthrough Therapy designations. Imbruvica also received Orphan Drug designation for this indication, which provides incentives to assist and encourage the development of drugs for rare diseases.

The FDA granted the approval of Imbruvica to Pharmacyclics LLC.