FDA accepts a supplemental new drug application for ruxolitinib in steroid-refractory cGvHD

On February 22, 2021, the U.S. Food and Drug Administration (FDA) granted priority review to a supplemental new drug application (sNDA) for ruxolitinib for the treatment of patients aged 12 years and older with steroid-refractory chronic graft-versus-host disease (cGvHD).¹

Ruxolitinib is a first-in-class JAK1/JAK2 inhibitor already approved by the FDA for the treatment of polycythemia vera and myelofibrosis in adults, and for steroid-refractory acute GvHD in adult and pediatric patients ≥ 12 years.

The sNDA submission was based on data from the REACH3 study (NCT03112603). This randomized, open-label, multicenter phase III study evaluated the safety and efficacy of ruxolitinib versus best available therapy (BAT) in patients with steroid-refractory cGvHD. The trial met its primary endpoint, with a significantly higher overall response rate observed for ruxolitinib compared with BAT. In addition, treatment with ruxolitinib was associated with longer median failure-free survival, significant symptom improvement, and a higher rate of best overall response compared to BAT. The safety profile was consistent with the known safety profile of ruxolitinib. Watch our interview with Robert Zeiser on the REACH3 results, here.

The Prescription Drug User Fee Act target action date for ruxolitinib in steroid-refractory cGvHD is June 22, 2021.¹