The U.S. Food and Drug Administration (FDA) approved Imbruvica (ibrutinib) for treatment of adult patients with chronic graft-versus-host-disease (cGVHD) who failed to respond to other forms of systemic therapy, AbbVie announced.
FDA’s decision means Imbruvica is the first and only therapy specifically approved for adults with cGVHD, a serious outcome that may arise after patients undergo stem cell or bone marrow transplants. This is an important decision for sickle cell anemia patients who may undergo stem cell transplant.
“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,” David Miklos, MD, PhD, said in a press release. Miklos is associate professor of medicine (Blood and Marrow Transplantation) at Stanford University.
“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,” Miklos added.
Approval followed the positive outcomes of patients in the ongoing Phase 1b/2 trial (NCT02195869), which is assessing Imbruvica’s safety and clinical effectiveness in 42 patients with cGVHD who failed to respond to corticosteroid therapy.
The results showed that during a median follow-up of 14 months, Imbruvica-treated patients had an overall response rate of 67%, from which 21% achieved a complete response. Moreover, 61% of responders experienced significant improvements in symptoms, and 62% reduced steroid dose. The trial is ongoing and final data collection for complete analysis is expected in December 2017.
“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,” said Lori Styles, MD, senior medical director and GVHD program clinical lead at Pharmacyclics LLC, an AbbVie company. “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,” she said.
FDA previously granted Imbruvica four breakthrough therapy designations, designed to expedite the development of therapeutics for serious and life-threatening diseases.