FDA Approves Imbruvica for Adults with Chronic Graft-Versus-Host-Disease

FDA Approves Imbruvica for Adults with Chronic Graft-Versus-Host-Disease

The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other lines of therapy. With this decision, Imbruvica, already approved for several forms of lymphoma, becomes the only therapy specifically approved for adults patients with cGVHD.

Pharmacyclics, AbbVie, and Janssen Biotech are the three partners involved in the development and commercialization of Imbruvica, a first-in-class Bruton’s tyrosine kinase (BTK) inhibitor.

“The FDA’s approval of Imbruvica in chronic graft-versus-host-disease after failure of one or more lines of systemic therapy addresses an area of high unmet medical need for patients and marks the first approved use for the therapy outside of blood cancers,” Lori Styles, MD, senior medical director and GVHD program clinical lead at Pharmacyclics, an AbbVie company, said in a press release.

“This approval is an indicator of what is possible with Imbruvica, and we remain excited about the clinical utility of Imbruvica in other disease areas. We continue to explore the full potential of this therapy and believe our comprehensive clinical trial program will help advance patient care,” Styles added.

The FDA’s decision was based on results from the ongoing PCYC-1129 Phase 1b/2 clinical trial, (NCT02195869), evaluating Imbruvica’s safety and effectiveness in 42 patients with cGVHD after they failed to respond to other therapies, including first-line corticosteroid therapy.

The most common diseases resulting in the need for transplant in the study were acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), and chronic lymphocytic leukemia (CLL).

The open-label, multicenter, single-arm trial showed that 67% of patients receiving Imbruvica responded to the treatment, including 21% complete responses. The rate of sustained response for at least 20 weeks was 48% for all patients. Of the patients treated with Imbruvica, 24% discontinued treatment due to adverse reactions, the most common being fatigue and pneumonia.

cGVHD remains a serious complication of stem cell or bone marrow transplant. The disease occurs in patients who have undergone stem cell or bone marrow transplant from partially matched donors. In cGVHD, donor immune cells attack the recipient’s tissues and organs.

Approximately 30-70% of post-transplant patients will develop cGVHD. The disease usually manifests a few months after a transplant and can last several years.

“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, MD, PhD, 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,” said Miklos, associate professor of medicine at Stanford University.

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