FDA Approves Ruxolitinib Tablets for Hematologic Malignancies

The FDA has approved ruxolitinib extended-release tablets (Jakafi XR) as a treatment for adults with intermediate- or high-risk myelofibrosis, adults with polycythemia vera with a poor response to or intolerance of hydroxyurea, for patients 12 years and older with steroid-refractory acute graft-versus-host disease (GVHD) or chronic GVHD following progression on 1 or 2 lines of systemic treatment, according to a press release from the developer, Incyte.¹

Supporting data for the approval came from a clinical study presented at the 2025 American Society of Hematology Annual Meeting & Exposition (ASH), which demonstrated the bioequivalence of ruxolitinib extended-release tablets vs reference ruxolitinib (Jakafi).² The extended-release tablets have demonstrated the potential for similar clinical benefit compared with the reference drug based on key measures of steady-state drug exposure.

Other studies of ruxolitinib across different patient populations have shown that the most common adverse effects include low platelet counts or red blood cell counts, bruising, dizziness, headache, and diarrhea in those with myelofibrosis or polycythemia vera. Additionally, other common toxicities include low platelet counts, low red or white blood cell counts, infections, and swelling in patients with acute GVHD as well as low red blood cell or platelet counts and infections among those with chronic GVHD.

“Patients living with chronic conditions like myeloproliferative neoplasms [MPNs] and GVHD often struggle with managing complex treatment regimens or have multiple conditions. Since its initial approval in 2011, ruxolitinib has helped transform the treatment landscape for patients with MPNs and GVHD,” Naveen Pemmaraju, MD, a professor of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, stated in the press release.¹ “With the approval of [extended-release ruxolitinib tablets], appropriate patients now have the choice of a single daily tablet.”

Developers designed this formulation of ruxolitinib, a first-in-class JAK1/JAK2 inhibitor, as once-daily extended-release tablets. According to the press release, it is not yet known if extended-release ruxolitinib tablets are effective or safe among children with myelofibrosis or polycythemia vera.

“The approval of [extended-release ruxolitinib tablets] reinforces Incyte’s leadership in hematology and our focus on meeting the evolving needs of patients with MPNs and GVHD. [Extended-release ruxolitinib tablets] offer appropriate patients and physicians a once-daily option, expanding choice without changing the well-established role of [ruxolitinib] in clinical practice,” Bill Meury, chief executive officer of Incyte, the developer of extended-release ruxolitinib tablets, said in the press release.¹

References

1. Incyte announces FDA approval of Jakafi XR™ (ruxolitinib) extended-release tablets for the treatment of myelofibrosis, polycythemia vera and graft-versus-host disease. News release. Incyte. May 1, 2026. Accessed May 4, 2026. https://tinyurl.com/3mpajbxx
2. Gong X, Xun Z, Getsy J, McGee R, Mondick J, Punwani N. Bioequivalence of ruxolitinib once-daily extended-release vs twice-daily immediate-release tablets in healthy adults. Blood. 2025;146(suppl 1):5045. doi:10.1182/blood-2025-5045