The Food and Drug Administration has approved Jakafi® (ruxolitinib) for the treatment of chronic graft-versus-host disease (GVHD) after failure of 1 or 2 lines of systemic therapy in patients 12 years of age and older.
The approval was based on data from the multicenter, randomized, open-label phase 3 REACH3 study (ClinicalTrials.gov Identifier: NCT03112603), which assessed the efficacy and safety of ruxolitinib, a Janus kinase (JAK1/JAK2) inhibitor, in 329 patients aged 12 years and older with steroid-refractory chronic GVHD after allogeneic stem cell transplantation.
Patients were randomly assigned 1:1 to receive either ruxolitinib 10mg twice daily or best available therapy (BAT; investigator selected: extracorporeal photopheresis, low-dose methotrexate, mycophenolate mofetil, everolimus, sirolimus, infliximab, rituximab, pentostatin, imatinib, or ibrutinib). Efficacy was based on overall response rate (ORR) through cycle 7 day 1, defined as the percentage of patients demonstrating a complete or partial response.
Results showed an ORR of 70% (95% CI, 63-77) for the ruxolitinib arm vs 57% (95% CI, 49-65) for the BAT arm. Among the responders, median time to first response was 3 weeks (range, 2 to 24) with ruxolitinib and 4 weeks (range, 2 to 25) with BAT. Median duration of response was 4.2 months (95% CI, 3.2-6.7) and 2.1 months (95% CI, 1.6-3.2) for the ruxolitinib and BAT arms, respectively. Median time from first response to death or new systemic therapies for chronic GVHD was 25 months (95% CI, 16.8-NE) for ruxolitinib and 5.6 months (95% CI, 4.1-7.8) for BAT.
As for safety, the most common hematologic adverse reactions (incidence greater than 35%) were anemia and thrombocytopenia. The most common nonhematologic adverse reactions (incidence 20% or greater) were infections (pathogen not specified) and viral infections.
Jakafi is also approved for the treatment of steroid-refractory acute GVHD, myelofibrosis, and polycythemia vera. The product is supplied as 5mg, 10mg, 15mg, 20mg, and 25mg tablets.
- Incyte announces FDA approval of Jakafi® (ruxolitinib) for treatment of chronic graft-versus-host disease (GVHD). News release. Incyte. Accessed September 22, 2021. https://www.businesswire.com/news/home/20210922005863/en/Incyte-Announces-FDA-Approval-of-Jakafi%C2%AE-ruxolitinib-for-Treatment-of-Chronic-Graft-Versus-Host-Disease-GVHD.
- Incyte announces positive phase 3 REACH3 study data published in NEJM for ruxolitinib (Jakafi®) in chronic graft-versus-host disease. News release. Incyte. July 14, 2021. Accessed September 22, 2021. https://investor.incyte.com/press-releases/press-releases/2021/Incyte-Announces-Positive-Phase-3-REACH3-Study-Data-Published-in-NEJM-for-Ruxolitinib-Jakafi-in-Chronic-Graft-Versus-Host-Disease/default.aspx.
- Jakafi. Package insert. Incyte; 2021. Accessed September 22, 2021. https://www.jakafi.com/pdf/prescribing-information.pdf.