FDA Approved First Treatment for Chronic GVHD
August 2, 2017
The US Food and Drug Administration (FDA) has expanded the approval of a therapy to provide the first option for chronic graft versus host disease (cGVHD).
Imbruvica (ibrutinib, Janssen Pharmaceuticals) is now approved to include adult patients with cGVHD after patients experienced failure of one or more treatments. This is the first therapy approved by the FDA for cGVHD, a condition estimated to occur in 30% to 70% of patients who receive hematopoietic stem cell transplantation.
Imbruvica is a kinase inhibitor that was previously approved for the treatment of chronic lymphocytic leukemia, Waldenström’s macroglobulinemia, and marginal zone lymphoma.
Approval for this new indication was based on single-arm trial that included 42 patients with cGVHD whose symptoms persisted despite treatment with corticosteroids. Overall, 67% of patients who received Imbruvica experienced symptom improvement, and 48% experienced improvements that lasted 5 months or longer.
Common adverse effects associated with Imbruvica included fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, stomatitis, nausea, hemorrhage, anemia, and pneumonia. Women who are pregnant or breastfeeding are not recommended to receive Imbruvica as the drug may harm developing fetuses or newborn babies.
In addition, serious adverse events included severe bleeding, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, and metabolic abnormalities.—Zachary Bessette