FDA Accepts for Review the Application of IMBRUVICA® for Chronic Graft-Versus-Host-Disease (cGVHD)

Janssen Research & Development, LLC announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for ibrutinib (IMBRUVICA^®) for the treatment of patients with chronic graft-versus-host-disease (cGVHD) after failure of one or more lines of systemic therapy. GVHD is a potential life-threatening consequence of stem cell or bone marrow transplant, with no approved treatments or established standards of care specifically indicated for the condition in patients who have failed initial steroid therapy. IMBRUVICA is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.

"Patients with chronic graft-versus-host-disease face an unpredictable, debilitating and sometimes life-threatening disease journey, which is further complicated by lack of FDA-approved medicines. We hope this filing acceptance and the robust body of evidence supporting ibrutinib in this condition will translate into a future indication in cGVHD and a new, much-needed treatment option," said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Development, Hematology for Janssen Research & Development, LLC. "We continue to study the mechanism of action of IMBRUVICA, and are committed to exploring its potential to transform treatment paradigms and patient outcomes beyond its current indications in blood cancers. Our investigation into cGVHD is one of a range of disease areas we are looking at outside of the hematologic malignancy category."

The sNDA is supported by data from a single-arm Phase 1b/2 trial (PCYC-1129) examining the safety and efficacy of ibrutinib in patients with cGVHD who have failed first-line corticosteroid therapy and require additional therapy. The data was accepted as a late-breaker and presented at the American Society of Hematology Annual Meeting in December 2016 and the Blood and Marrow Transplantation Tandem Meeting in February 2017. Based on this data, a Phase 3 study was initiated to evaluate ibrutinib with corticosteroid versus placebo with corticosteroid as a first-line therapy for patients with new onset moderate or severe cGVHD; the trial is currently ongoing. The FDA granted Breakthrough Therapy Designation and Orphan Drug Designation in June 2016 for ibrutinib as a potential treatment for cGVHD after failure of one or more lines of systemic therapy.

GVHD is a medical complication of allogeneic stem cell or bone marrow transplant and can be a serious and debilitating condition. The condition occurs when donor immune cells attack the patients' tissues and it can affect almost any organ in the body. The incidence of cGVHD has continued to increase over time. In fact, approximately 30-70 percent of post-allogeneic transplant patients develop cGVHD.