The Food and Drug Administration (FDA) has approved ruxolitinib (Jakafi)  to treat chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy in patients 12 years and older.

Chronic GVHD is a rare complication that can occur after a stem cell or bone marrow transplant. Symptoms may include skin rash, mouth sores, dry eyes, liver inflammation, development of scar tissue in the skin and joints, and damage to the lungs. The exact cause of chronic GVHD is unknown but likely due to a complex immune-mediated interaction between the donor and recipient cells. Chronic GVHD is usually treated with prednisone or other similar anti-inflammatory or immunosuppressive medications but not all patients respond to or can tolerate that treatment.

The approval of ruxolitinib was largely based on the REACH-3 trial (NCT03112603), a randomized, open-label, multicenter clinical trial comparing ruxolitinib to best available therapy (BAT) for corticosteroid-refractory chronic GVHD after allogeneic stem cell transplantation (N=329).

The trial observed that overall response rate (ORR) was 70% for the ruxolitinib arm vs 57% for the BAT arm. The median durations of response was 4.2 months for the ruxolitinib arm vs 2.1 months for the BAT arms. The median times from first response to death or new systemic therapies for chronic GVHD were 25 months for the ruxolitinib arm vs 5.6 months for the BAT arms.

The most common hematologic adverse reactions with ruxolitinib were anemia and thrombocytopenia and the most common nonhematologic adverse reactions were infections and viral infection.

The recommended ruxolitinib starting dose for chronic GVHD is 10 mg given orally twice daily.

This is the second orphan drug approved for chronic GVHD this year. In July, the FDA approved belumosudil for chronic GVHD. Both ruxolitinib and belumosudil are kinase inhibitors.

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