The Food and Drug Administration (FDA) has approved Jakafi (ruxolitinib; Incyte) for treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adult and pediatric patients ≥12 years old.

The approval was based on data from the REACH1 trial, an open label, single arm, study conducted in 71 patients in which 49 patients were refractory to steroids alone. The efficacy of Jakafi was based on Day 28 overall response rate (ORR) (defined as complete response, very good partial response or partial response by Center for International Blood and Marrow Transplant Research [CIBMTR] criteria) and the duration of response.

Results showed that on Day 28, ORR was 57.1% among the 49 steroid-refractory patients; ORR was 100% for Grade 2 GVHD, 40.7% for Grade 3 GVHD, and 44.4% for Grade 4 GVHD. The median duration of response (calculated from Day 28 response to progression, new salvage therapy for acute GVHD or death from any cause) was 16 days. In addition, the median time from Day 28 response to either death or need for new therapy for acute GVHD (additional salvage therapy or increase in steroids) was 173 days.

With regard to safety, the most common adverse reactions among the 71 patients in the study were anemia, thrombocytopenia, neutropenia, infections and edema.

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“For the first time, patients with steroid-refractory acute GVHD, and the physicians that treat them, have an FDA-approved treatment for this serious disease,” stated Hervé Hoppenot, Chief Executive Officer, Incyte.

Jakafi, a Janus Associated Kinase (JAK)1/JAK2 inhibitor, is also approved for the treatment of myelofibrosis and polycythemia vera. The treatment is available in 5mg, 10mg, 15mg, 20mg, and 25mg tablets.

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