FDA Approves Jakafi for GVHD

This is the fourth approval for Jakafi, a JAK1/JAK2 inhibitor.

The FDA has approved Incyte’s Jakafi (ruxolitinib) for treatment of chronic graft-versus-host disease (GVHD) after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.

Graft-versus-host disease is a systemic disorder that occurs when the graft's immune cells recognize the host as foreign and attack the recipient’s body cells. It can occur in up to half of patients receiving hematopoietic stem cell transplantation.

“In the United States, there are over 14,000 people living with chronic GVHD, many of whom face significant complications that may impair daily activities and linger for years,” Susan Stewart, executive director, BMT InfoNet, said in a statement. “The approval of Jakafi is an exciting development for the GVHD community and an important step forward in the treatment of a disease with few options.” The Blood & Marrow Transplant Information Network (BMT InfoNet) is a leading advocacy organization for bone marrow, stem cell and cord blood transplant patients.

The approval was based on the REACH3 study, a phase 3 study of Jakafi in comparison to best available therapy for treatment of steroid-refractory chronic GVHD after allogeneic stem cell transplantation. The primary end point of overall response rate at week 24 was 49.7% for Jakafi compared with 25.6% for best available therapy. The most common nonhematologic adverse reactions (incidence ≥ 20%) were infections. Full results from the REACH3 study were published in the New England Journal of Medicine.

This approval is the fourth FDA-approved indication for Jakafi, which received FDA-approval in 2019 for steroid-refractory acute GVHD in adult and pediatric patients 12 years and older.

Jakafi is marketed by Incyte in the United States and by Novartis as Jakavi outside the United States.