FDA approves Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft-versus-host-disease (cGVHD) . AbbVie + Janssen Biotech, Inc.

AbbVie , announced the FDA has approved Imbruvica (ibrutinib) for the treatment of adult patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy. With this approval, Imbruvica becomes the first and only therapy specifically approved for adults with cGVHD, a serious and debilitating potential consequence of stem cell or bone marrow transplant.Imbruvica, a first-in-class Bruton's tyrosine kinase (BTK) inhibitor, is jointly developed and commercialized by Pharmacyclics LLC, an AbbVie company, and Janssen Biotech, Inc.

"Stem cell and bone marrow transplants can be life-saving treatment options for people with blood cancers or marrow failure syndromes; however, nearly half of transplant patients subsequently develop chronic graft-versus-host-disease, or cGVHD, in which the donor's immune cells damage the patient's normal organs and their quality of life," said David Miklos, M.D., Ph.D., Associate Professor of Medicine (Blood and Marrow Transplantation), Stanford University, and lead investigator of the Imbruvica cGVHD clinical study. "With Imbruvica, we observed sustained responses lasting five months or longer across multiple organs affected by this debilitating condition for 48 percent of all patients. This approval represents a major advance and provides physicians with a new option for adults with steroid refractory cGVHD."

The approval in cGVHD is based on findings from an open-label, multi-center, single-arm Phase 1b/II trial (PCYC-1129), which evaluated the safety and efficacy of ibrutinib in 42 patients with cGVHD that failed to respond to first-line corticosteroid therapy and required additional therapy (median age of 56; range: 19 to 74 years old; 52 percent male; 93 percent Caucasian). The most common underlying malignancies leading to transplantation for the study population were acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML) and chronic lymphocytic leukemia (CLL). The median time since cGVHD diagnosis was 14 months, and the median number of prior cGVHD treatments was two (range: one to three treatments). The majority of patients (88 percent) had at least two organs affected at baseline, including mouth (86 percent), skin (81 percent) and gastrointestinal tract (33 percent).