Ibrutinib (Imbruvica, Pharmacyclics LLC) has secured a new indication that makes it the first FDA-approved therapy for the treatment of chronic graft versus host disease (cGVHD).
The FDA expanded ibrutinib’s approval to include the treatment of adults with cGVHD after failure of one or more treatments. This life-threatening condition can occur after patients receive hematopoietic stem cell transplantation (HSCT) to treat certain blood or bone marrow cancers. In cGVHD, cells from the stem cell transplant attack healthy cells in a patient’s tissues. Symptoms of cGVHD can occur in the skin, eyes, mouth, gut, liver, and lungs. The condition is estimated to occur in 30% to 70% of all patients who receive HSCT.
“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.
The efficacy and safety of ibrutinib 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% of patients had two or more organs affected by cGVHD. In the trial, 67% of patients experienced improvements in their cGVHD symptoms. In 48% of patients in the trial, the improvement of symptoms lasted for up to five months or longer.
Common side effects of ibrutinib in patients with cGVHD include fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, stomatitis, nausea, hemorrhage, anemia, and pneumonia. Serious side effects include hemorrhage, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, and tumor lysis syndrome. Women who are pregnant or breastfeeding should not take ibrutinib because it may cause harm to a developing fetus or a newborn baby.
Ibrutinib, 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 indication priority review, breakthrough therapy, and orphan drug designations.
Source: FDA; August 2, 2017.