Imbruvica Becomes 1st FDA-Approved Therapy for Graft-Versus-Host-Disease in Adults

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by Alice Melão |

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cGVHD Imbruvica FDA

The U.S. Food and Drug Administration (FDA) has approved Imbruvica (ibrutinib) for the treatment of chronic graft-versus-host-disease (cGVHD), a common complication of stem cell or bone marrow transplant, in adult patients who failed to respond to other treatments.

The FDA approval makes Imbruvica the first and only medicine specifically approved for cGVHD in adults.

“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.

Imbruvica was designed to target the Bruton’s tyrosine kinase (BTK), which modulates the response of immune B-cells. It was jointly developed and commercialized by Pharmacyclics and Janssen Biotech.

In addition to cGVHD, Imbruvica also is used to treat several B-cell malignancies, including mantle cell lymphoma, Waldenström’s macroglobulinemia, and marginal zone lymphoma.

The FDA’s approval was based on positive data from the Phase 1b/2 PCYC-1129 trial (NCT02195869) in patients with cGVHD who failed to respond to first-line corticosteroid therapy.

The researchers observed that 67% of Imbruvica-treated patients improved their cGVHD symptoms affecting the skin, mouth, gastrointestinal tract, or liver. Of these, 21% completely responded and 45% partially responded. Overall, response rates were sustained for at least 20 weeks in 48% of patients.

More than 20% of patients receiving Imbruvica had adverse side effects, including fatigue, bruising, nausea, diarrhea, low platelet and red blood cell levels, and pneumonia. Twenty-four percent of patients discontinued treatment due to adverse reactions, mainly fatigue and pneumonia.

“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, associate professor of medicine at Stanford University, and lead investigator of the Imbruvica cGVHD clinical study.

“This approval represents a major advance and provides physicians with a new option for adults with steroid refractory cGVHD,” Miklos added.