FDA approves Incyte’s Jakafi XR for myelofibrosis, polycythemia vera, and graft-versus-host disease

Incyte has received US FDA approval for Jakafi Xr (ruxolitinib) extended-release tablets, a once-daily reformulation of its established JAK1/JAK2 inhibitor, across three hematologic indications: intermediate- or high-risk myelofibrosis, polycythemia vera in patients with inadequate response to or intolerance of hydroxyurea, and steroid-refractory acute or chronic graft-versus-host disease in adults and pediatric patients aged 12 and older.

The approval covers the same indications as the original twice-daily Jakafi (ruxolitinib), first approved in 2011, and does not alter the clinical scope of the franchise. Instead, it introduces a 55 mg extended-release tablet taken once daily, which the company said is bioequivalent to a 25 mg immediate-release tablet taken twice daily based on steady-state drug exposure measures. Jakafi Xr was made available for pharmacy orders by May 8, 2026.

The regulatory basis for approval was a bioequivalence study, presented as a poster at the American Society of Hematology annual meeting in 2025 by Gong et al., demonstrating that the pharmacokinetic profile of the once-daily formulation was comparable to the twice-daily reference product. No new efficacy trials were conducted; the safety profile of Jakafi Xr is established from the existing body of controlled studies across myelofibrosis, polycythemia vera, and both acute and chronic graft-versus-host disease. Common adverse reactions across indications include cytopenias, infections, bruising, dizziness, headache, and diarrhea, consistent with the known profile of the immediate-release formulation.

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The approval adds a dosing option within an established treatment framework rather than expanding ruxolitinib into new disease territory. For patients managing chronic conditions such as myeloproliferative neoplasms or graft-versus-host disease, a reduction from twice-daily to once-daily dosing may carry practical relevance, particularly where polypharmacy or adherence is a concern. The clinical equivalence claim rests on pharmacokinetic data rather than direct outcomes comparisons, and no head-to-head trial against the immediate-release tablet on efficacy endpoints was reported.

The myelofibrosis treatment landscape has evolved considerably since ruxolitinib’s initial approval, with several agents now approved alongside it. Pacritinib (Vonjo, CTI BioPharma) and fedratinib (Inrebic, Bristol Myers Squibb) are both approved for myelofibrosis and provide options for patients with severe thrombocytopenia or intolerance to ruxolitinib. In the GVHD space, belumosudil (Rezurock, Sanofi/Kadmon) received approval for chronic GVHD in 2021, and axatilimab (Niktimvo, Syndax Pharmaceuticals) was approved for the same indication in August 2024, reflecting a diversifying treatment landscape beyond JAK inhibition. The Jakafi Xr approval does not directly compete with these agents on mechanism but offers a formulation alternative within the ruxolitinib-treated patient population.


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