FDA Approvals of Systemic Therapy for Steroid-Refractory cGVHD

Second-Line Therapy and FDA Approvals

This segment reviews FDA-approved systemic therapies for chronic GVHD:

• Ibrutinib (2017): First approval for chronic GVHD after failure of ≥1 line of therapy
• Belumosudil (July 2021): Approved for chronic GVHD after ≥2 prior lines in patients ≥12 years
• Ruxolitinib (September 2021): Approved for chronic GVHD after 1-2 lines in patients ≥12 years
• Axatilimab (August 2024): Approved for chronic GVHD after ≥2 prior lines in adults and pediatric patients ≥40 kg

The panel discusses the long-term follow-up data from the ibrutinib trial, showing 69% overall response rate with higher efficacy in inflammatory features and moderate efficacy in sclerotic features. Common grade 3+ adverse events included pneumonia, fatigue, and diarrhea, with 43% discontinuation due to adverse effects.