August 2, 2017 -- The U.S. Food and Drug Administration today 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 landmark approval makes Imbruvica the first FDA-approved therapy specifically indicated for the treatment of cGVHD, addressing a critical unmet medical need for patients with this life-threatening condition.
About Chronic Graft Versus Host Disease (cGVHD)
cGVHD is a life-threatening complication that can arise after patients receive a hematopoietic stem cell transplantation (HSCT)—a stem cell transplant from blood or bone marrow—used to treat certain blood or bone marrow cancers. The condition occurs when the transplanted stem cells attack the healthy cells in the patient’s tissues, with symptoms potentially affecting the skin, eyes, mouth, gut, liver, and lungs. It is estimated that 30-70 percent of all patients who undergo HSCT will develop cGVHD.
FDA Perspective on the Approval
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, highlighted the significance of the approval: “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. 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.”
Clinical Trial: Efficacy and Safety Data
The approval of Imbruvica for cGVHD is based on data from a single-arm trial involving 42 patients with cGVHD whose symptoms persisted despite standard corticosteroid treatment. Most patients in the trial experienced symptoms such as mouth ulcers and skin rashes, and more than half had cGVHD affecting two or more organs. The results showed that 67 percent of patients experienced improvements in their cGVHD symptoms, and for 48 percent of these patients, the symptom improvement lasted for five months or longer.
Side Effects of Imbruvica for cGVHD
Common side effects of Imbruvica in patients with cGVHD include fatigue, bruising, diarrhea, thrombocytopenia (low blood platelets), muscle spasms, swelling, stomatitis (mouth sores), nausea, hemorrhage (severe bleeding), anemia (low red blood cells), and pneumonia (lung infection).
Serious side effects of Imbruvica include severe bleeding (hemorrhage), infections, cytopenias (low blood cells), atrial fibrillation (irregular heartbeat), hypertension (high blood pressure), second primary malignancies (new cancers), and tumor lysis syndrome (metabolic abnormalities). Importantly, women who are pregnant or breastfeeding should not take Imbruvica, as it may cause harm to a developing fetus or newborn baby.
Imbruvica’s Previous Approvals and FDA Designations
Imbruvica, a kinase inhibitor, had previously been approved for certain indications in the treatment of chronic lymphocytic leukemia, Waldenström’s macroglobulinemia, and marginal zone lymphoma, as well as under accelerated approval status for mantle cell lymphoma.
For this cGVHD indication, the FDA granted the application Priority Review and Breakthrough Therapy designations. Imbruvica also received Orphan Drug designation, which provides incentives to support the development of drugs for rare diseases. The FDA granted the approval of Imbruvica to Pharmacyclics LLC.
