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New Treatment for Acute Graft-Versus-Host Disease

Only FDA-Approved Therapy for GVHD in Adults and Children

The FDA has approved ruxolitinib (Jakafi®, Incyte Corporation) for the treatment of steroid-refractory acute graft-versus-host disease (GVHD) in adults and pediatric patients aged 12 years and older.

Ruxolitinib is also indicated for treating polycythemia vera in adults with an insufficient response to or intolerance of hydroxyurea, and in adults with intermediate/high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF.

GVHD, which can occur after an allogeneic stem cell transplant, has two major forms: acute and chronic. It can affect multiple organ systems including the skin, gastrointestinal tract, and liver. Patients who develop steroid-refractory acute GVHD can develop severe disease, and one-year mortality rates are approximately 70%.

Approval was based on data from REACH1, an open-label, single-arm, multicenter study of ruxolitinib in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD.

The most frequently reported adverse reactions among all 71 participants were infections (55%) and edema (51%). The most common laboratory abnormalities were anemia, thrombocytopenia, and neutropenia.

The REACH program also includes the collaborative, randomized pivotal phase 3 trials in patients with steroid-refractory acute GVHD (REACH2) and steroid-refractory chronic GVHD (REACH3). Results from both trials are expected before the end of 2019.

Incyte plans to make ruxolitinib available immediately to appropriate patients with steroid-refractory acute GVHD. 

Source: Incyte, May 24, 2019

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