Recently, the European Medicines Agency's Committee on Medical Products for Human Use recommended the approval of ruxolitinib (Jakafi) for the treatment of patients aged 12 years or older with acute or chronic graft-versus-host disease (GVHD) who have had an inadequate response to corticosteroids or other systemic therapies.
Based on findings from the Phase 3 trials REACH2 (NCT02913261) and REACH3 (NCT03112603), in which the drug demonstrated superiority over best available therapy (BAT) in steroid-refractory and dependent acute and chronic GVHD patients, respectively.
Specifically, in patients with steroid-refractory or dependent acute GVHD, ruxolitinib was found to produce an overall response rate (ORR) of 62.3% on day 28, compared to 39.4% for BAT. At 24 weeks, ruxolitinib also demonstrated a superior ORR compared to BAT in patients with steroid-refractory or dependent chronic GVHD, with incidences of 49.7% and 25.6%, respectively.
The European Commission recommends ruxolitinib (Jakafi) for acute or chronic graft-versus-host disease.
“For many blood disorders, allogeneic transplantation is the only potentially curative treatment; however, half of those who undergo it will develop acute or chronic GVHD,” said Dr. Robert Zeiser of the Department of Hematology, Oncology and Stem Cell Transplantation at Freiburg University Hospital in a press release. “Encouragingly, we may soon be able to provide a new standard of care for patients with other debilitating conditions who do not respond adequately to first-line corticosteroids or other systemic therapies.”
Ruxolitinib is the active pharmaceutical ingredient in Incyte’s oral drug Jakafi, which has been approved in the United States for three indications: (1) for the treatment of adult patients with polycythemia vera (PV) who have an inadequate or intolerable response to thioglycosuria; (2) for the treatment of adult patients with intermediate- to high-risk myelofibrosis (MF), including primary MF, post-PV MF, and post-essential thrombocythemia MF; and (3) for the treatment of patients with steroid-refractory acute graft-versus-host disease (GVHD). The third indication was approved by the FDA in May 2019, making it the first drug approved for this indication.
