The U.S. Food and Drug Administration (FDA) has approved Imbruvica (ibrutinib) for treating adults with chronic graft versus host disease (cGVHD) after failure of one or more treatments.
This is the first FDA-approved treatment for cGVHD.
cGVHD is a life-threatening condition that may develop in patients after they receive a hematopoietic stem cell transplantation (HSCT) to treat various blood and marrow cancers.
cGVHD develops as a result of cells from the transplant attacking the recipients own cells. It is estimated to occur in 30-70% of 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, M.D., 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 in a news release
. “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 approval was based on a single-arm trial of 42 patients with cGVHD whose symptoms remained after receiving standard treatment with corticosteroids. In the trial, 67% of patients experienced improvements in their cGVHD symptoms and almost half of the patients (48%) had improvements that lasted 5 months or longer.
Common side effects include fatigue, bruising, diarrhea, thrombocytopenia, muscle spasms, stomatitis, nausea, severe bleeding, anemia, and pneumonia.
Serious side effects include severe bleeding, infections, cytopenias, atrial fibrillation, hypertension, second primary malignancies, and tumor lysis syndrome. Women who are pregnant or breastfeeding should not take Imbruvica.
Imbruvica is a kinase inhibitor that is also approved for a number of rare cancers, including chronic lymphocytic leukemia, Waldenström’s macroglobulinemia and marginal zone lymphoma.
The drug is manufactured by Pharmacyclics.
For more up-to-date information of FDA approvals, applications and designations, follow Rare Disease Report