FDA Approves Once-Daily Jakafi XR for Myelofibrosis and Other Conditions

The U.S. Food and Drug Administration (FDA) has approved Jakafi XR (ruxolitinib) extended-release tablets for the treatment of several chronic blood conditions and transplant complications in adult and pediatric populations.

This new, once-daily formulation is authorized for use in adults with intermediate- or high-risk myelofibrosis (MF) and adults with polycythemia vera (PV) who have not responded well to or cannot tolerate hydroxyurea. Additionally, the approval extends to adults and children aged 12 years and older who are experiencing steroid-refractory acute graft-versus-host disease (GVHD) or chronic GVHD after the failure of one or two lines of systemic therapy.

By providing a single daily dose, the manufacturer aims to offer a new option for patients who may struggle with managing complex treatment regimens while living with rare, chronic blood cancers and immune responses following stem cell transplants.

Main data that support the findings

The FDA based its approval on clinical data demonstrating that the extended-release formulation provides the same level of medication to the body as the original version. Specifically, the study showed that a single 55 mg Jakafi XR tablet taken once-daily is bioequivalent to a single 25 mg Jakafi tablet taken twice-daily.

Bioequivalence means that the drug delivers the same active ingredient at comparable levels throughout the day based on key measures of steady-state drug exposure. Because the exposure levels are comparable, the once-daily tablet indicates the potential for a similar clinical benefit to the twice-daily immediate-release dosage form. This pharmacokinetic data supports the transition to a simplified dosing schedule without changing the established role the medication plays in clinical practice.

For patients living with myeloproliferative neoplasms (MPNs) like MF and PV, or those managing GVHD, the availability of a once-daily tablet addresses the evolving needs of those who often manage multiple conditions simultaneously. Researchers noted that since the initial approval of Jakafi in 2011, the therapy has been a standard in the treatment landscape, and this new formulation expands choice for appropriate patients and their physicians.

Trial details

The approval process relied on a clinical study designed to compare the performance of the extended-release 55 mg tablet against the 25 mg immediate-release tablet. The trial focused on steady-state drug exposure to ensure that the medication remains consistent in the patient's system over a 24-hour period.

While the bioequivalence study was the primary driver for this specific formulation's approval, the broader clinical utility of the active ingredient is supported by long-standing research. Myelofibrosis is a rare, chronic blood cancer where bone marrow defects lead to abnormal blood cell production and scar tissue formation. This can result in severe anemia, weakness and an enlarged spleen. Polycythemia vera is a similar chronic blood cancer where the marrow produces too many red blood cells, white blood cells and platelets, which can thicken the blood and increase the risk of clotting complications like stroke or heart attack.

In the context of GVHD, the medication is used after an allogeneic stem cell transplant, where donated cells initiate an immune response against the recipient's organs. The trials for this condition include both acute GVHD, which typically occurs within 100 days of a transplant, and chronic GVHD, which generally occurs after that 100-day window. Both forms can impact the skin, digestive tract and liver.

Safety

The safety profile for Jakafi XR has been established through adequate and well-controlled studies of the immediate-release version in adult patients with MF and PV, as well as adult and pediatric patients with acute and chronic GVHD.

Common side effects identified in these studies vary depending on the condition being treated. For patients with MF and PV, the most frequent side effects include:

• Low platelet counts
• Low red blood cell counts
• Bruising
• Dizziness
• Headache
• Diarrhea

For patients managing acute GVHD, common reactions include low platelet counts, low red or white blood cell counts, infections, and swelling. In cases of chronic GVHD, the most common side effects reported were low red blood cell or platelet counts and infections, including viral infections.

It is important to note that while the medication is approved for children 12 years and older with GVHD, it is not known if Jakafi XR is safe or effective in children for the treatment of myelofibrosis or polycythemia vera. Patients prescribed the new formulation have access to support programs like IncyteCARES to help navigate reimbursement, education and financial assistance.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI, reviewed by a human editor, but not independently verified by a medical professional.

Reference: “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.

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