The U.S. Food and Drug Administration ( FDA ) has approved Jakafi ( Ruxolitinib ) for the treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.
Jakafi is the first and only FDA-approved treatment for this indication.
The approval was based on data from REACH1, an open-label, single-arm, multicenter study of Jakafi in combination with corticosteroids in patients with steroid-refractory grade II-IV acute GVHD.
Of the 71 patients recruited into REACH1, 49 patients were refractory to steroids alone, 12 patients had received two or more prior anti-GVHD therapies and 10 patients did not otherwise meet the FDA definition of steroid-refractory.
Jakafi was administered at 5 mg twice daily, and the dose could be increased to 10 mg twice daily after three days in the absence of toxicity.
The efficacy of Jakafi was evaluated based upon day 28 overall response rate ( ORR ), defined as a complete response ( CR ), very good partial response or partial response based on the Center for International Blood and Marrow Transplant Research ( CIBMTR ) criteria.
The day 28 ORR in the 49 patients refractory to steroids alone was 57% with a CR rate of 31%.
The most frequently reported adverse reactions among all 71 study participants were infections ( 55% ) and edema ( 51% ), and the most common laboratory abnormalities were anemia ( 75% ), thrombocytopenia ( 75% ) and neutropenia ( 58% ).
GVHD is a condition that can occur after an allogeneic stem cell transplant where the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality.
There are two major forms of GVHD, acute and chronic, that can affect multiple organ systems including the skin, gastrointestinal tract and liver.
Patients who develop steroid-refractory acute GVHD can progress to severe disease, with one-year mortality rates of approximately 70%.
Jakafi is a first-in-class JAK1/JAK2 inhibitor approved by the U.S. FDA for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.
Jakafi is also indicated for treatment of polycythemia vera in adults who have had an inadequate response to or are intolerant of Hydroxyurea as well as intermediate or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis and post-essential thrombocythemia myelofibrosis in adults. ( Xagena )
Source: Incyte, 2019