National Stroke Foundation

Jakafi (ruxolitinib)

The following drug information is obtained from various newswires, published medical journal articles, and medical conference presentations.

Company:

Approval Status:

Approved May 2019

Specific Treatments:

steroid-refractory acute graft-versus-host disease in adults and pediatrics >12 years

General Information

Jakafi (ruxolitinib) is a kinase inhibitor.

Jakafi is specifically indicated for steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older.

Jakafi is supplied as a tablet for oral administration. Doses should be individualized based on safety and efficacy. The starting dose of Jakafi for acute graft-versus-host disease is 5 mg given orally twice daily. 

Clinical Results

FDA Approval

The FDA approval of Jakafi for acute graft-versus-host disease was based on 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 percent with a CR rate of 31 percent.

 

Side Effects

Adverse effects associated with the use of Jakafi may include, but are not limited to, the following:

infections

edema

anemia

thrombocytopenia

neutropenia

Mechanism of Action

Jakafi (ruxolitinib) inhibits Janus Associated Kinases (JAKs) JAK1 and JAK2 which mediate the signaling of a number of cytokines and growth factors that are important for hematopoiesis and immune function. JAK signaling involves recruitment of STATs (signal transducers and activators of transcription) to cytokine receptors, activation and subsequent localization of STATs to the nucleus leading to modulation of gene expression. 

Additional Information

For additional information regarding Jakafi or steroid-refractory acute graft-versus-host disease in adults and pediatrics >12 years, please visit the Jakafi page