FDA approves Imbruvica as first therapy for chronic graft-versus-host disease
The FDA approved ibrutinib for the treatment of adults with chronic graft-versus-host disease who failed prior systemic therapy.
Ibrutinib (Imbruvica; Pharmacyclics, Janssen) — a Bruton’s tyrosine kinase inhibitor already indicated for treatment of certain patients with leukemia, lymphoma and Waldenstrom’s macroglobulinemia — is the first therapy specifically approved to treat chronic GVHD.
“Patients with chronic GVHD 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,” 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, said in a press 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 FDA based the approval in part on results from the open-label, multicenter, single-arm PCYC-1129-CA trial, designed to evaluate the efficacy and safety of 420 mg ibrutinib once daily for 42 patients with chronic GVHD who failed first-line corticosteroid therapy and required additional therapy.
Most patients (88%) had at least two organs involved at baseline, the most common of which were the mouth (86%), skin (81%) and gastrointestinal tract (33%).
Twenty-eight patients (67%; 95% CI, 51-80) achieved a response, with median time to response of 12.3 weeks. Researchers observed responses in all organs involved.
Twenty patients (48%) experienced symptom improvement for 5 months or longer.
The most common adverse reactions included fatigue, bruising, diarrhea, thrombocytopenia, stomatitis, muscle spasms, nausea, hemorrhage, anemia and pneumonia.
One patient experienced atrial fibrillation.