The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to the tyrosine kinase inhibitor ibrutinib for the treatment of patients with chronic graft-versus-host disease (cGVHD) after failure of one or more lines of systemic therapy. The FDA also granted the therapy Orphan Drug Designation for cGVHD.
The FDA’s decision was based on data from a phase Ib/II study of 28 patients with steroid-dependent or refractory cGVHD who received single-agent ibrutinib. The study’s primary endpoint was reduction in cGVHD, according to National Institutes of Health (NIH) Consensus Response Criteria. The overall response rate was 55 percent.
The most common all-grade adverse events (AEs) in the trial were fatigue (50%), bruising (25%), diarrhea (25%), and nausea (21%). The most frequently observed grade ≥3 AEs were fatigue, which occurred in five patients, and diarrhea, pneumonia, and headache (2 patients each). Six patients discontinued therapy due to an AE, and three patients discontinued after developing progressive cGVHD.
Source: AbbVie press release, June 29, 2016.