Policy & Regulation
US FDA Approves Jakafi for Treatment of Chronic Graft-Versus-Host Disease
22 September 2021 - - The US Food and Drug Administration has approved Jakafi (ruxolitinib) for treatment of chronic graft-versus-host disease after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older, US-based biopharmaceutical company Incyte (NASDAQ: INCY) said.

The FDA approval was based on the REACH3 study, a Phase 3, randomized, open-label, multicenter study of Jakafi in comparison to best available therapy for treatment of steroid-refractory chronic GVHD after allogeneic stem cell transplantation.

The primary endpoint of overall response rate at Week 24 (i.e., Cycle 7 Day 1) was 49.7% for Jakafi compared to 25.6% for BAT (P 35%) were anemia and thrombocytopenia. The most common nonhematologic adverse reactions (incidence ≥ 20%) were infections (pathogen not specified) and viral infection.

Full results from the REACH3 study were published in the New England Journal of Medicine.

GVHD is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient's organs.

There are two major forms of GVHD: acute, which generally occurs within 100 days of transplant, and chronic, which generally occurs more than 100 days after transplant3. Both forms are associated with significant morbidity and mortality and can affect multiple organ systems.

Jakafi's supplemental New Drug Application in chronic GHVD was reviewed under the FDA's Priority Review program as well as the Project Orbis program, an initiative of the FDA Oncology Center of Excellence that provides a framework for concurrent submission and review of oncology drugs among its international partners.

Participating countries for this application include Canada, Australia, Switzerland, Brazil and the United Kingdom.

REACH3 (NCT03112603), a randomized, open-label, multicenter Phase 3 study co-sponsored by Novartis and Incyte, evaluated the safety and efficacy of ruxolitinib compared with best available therapy in patients with steroid-refractory chronic GVHD.

Jakafi is a JAK1/JAK2 inhibitor approved by the US FDA for treatment of chronic GVHD after failure of one or two lines of systemic therapy 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, intermediate or high-risk myelofibrosis, including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults, and for treatment of steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is marketed by Incyte in the US and by Novartis as Jakavi (ruxolitinib) outside the US Jakafi is a registered trademark of Incyte. Jakavi is a registered trademark of Novartis AG in countries outside the US.

Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics.