The Potential Benefits of Once-Daily Extended-Release Ruxolitinib Tablets

Naveen Pemmaraju, MD, spoke with CancerNetwork^® about the potential clinical implications surrounding the FDA approval of extended-release ruxolitinib tablets (Jakafi XR) for patients with myelofibrosis, polycythemia vera, or graft-versus-host disease. Specifically, he highlighted the potential benefits this tablet formulation may offer compared with reference ruxolitinib (Jakafi) across these patient populations.

The once-daily extended-release tablets, Pemmaraju said, may address a variety of issues associated with administering reference ruxolitinib on a twice-daily basis. For example, the newly approved formulation may help patients avoid intentional or unintentional adherence, in which they do not receive the second dose of ruxolitinib due to various external circumstances. Additionally, the extended-release tablets may prevent the occurrence of what Pemmaraju described as “ruxolitinib withdrawal syndrome”, which may cause patients to miss the second or subsequent doses of ruxolitinib in the coming days.

Looking ahead, Pemmaraju noted a need for real-world data on whether the once-daily formulation may reduce drug-to-drug interactions and consequently affect treatment absorption compared with twice-daily dosing.

Pemmaraju is a Professor in the Department of Leukemia in the Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, where he also serves as the co-head of the MPN Section, the program director for BPDCN, and the executive director for the MDACC Network.

Transcript:

In this approval, which is a bioequivalence approval, it’s trying to show that, even though our prior drug was [given] twice a day, here in the new formulation, [once-daily treatment] can potentially have [similar] efficacy and no new safety signals. That’s what we’re hoping for as it goes live post-FDA approval.

The issue for a lot of my patients is the twice-a-day dosing. The first dose, oftentimes [patients] may remember to take; usually, it’s in the morning, with the daily activities. But sometimes at night or with travel, weekends, holidays, change in work or personal travel, and other shifting schedules, the second dose can be missed unintentionally.Let’s call that unintentional adherence issues.

In unintentional nonadherence, the patient intends to take the dose as prescribed—in this case, twice a day for, let’s say, a month for a cycle. But due to travel, emergencies, or any number of reasons, [they] ultimately miss a dose. That, we believe, is probably common in all chronic oral drug regimens in general. But then there’s also intentional nonadherence, which means that for whatever reason—maybe a patient is feeling toxicities—and without reaching out to the healthcare team, they self-discontinue. That’s one example. For other reasons, a patient [may have been] unable to take the drug. Maybe the drug isn’t able to get refilled due to some insurance or reimbursement issue. A lot of times, [patients] may end up rationing the drug or missing the second daily dose, etc. When you have a once-a-day formulation, it possibly could help that adherence factor, so unintentional and intentional adherence might be improved.

The second aspect is that of ruxolitinib withdrawal syndrome. There is such a thing as this type of a withdrawal syndrome. Sometimes, it can occur when the first dose is taken, and perhaps the second or subsequent doses are missed into the coming days. Perhaps, if you have a once-a-day formulation in those patients where there was a possibility of missing that second dose, it may prevent that. That could be something.

Finally, the other issue, which remains to be seen, is an unanswered medical question; we will have to see that in our own real-world analysis. Could there be fewer drug-to-drug interactions with once-a-day vs twice-a-day [dosing like] better absorption and fewer interactions with new medicines that are being prescribed? Of course, the flip side could also happen. There could be more [interactions]. That’s an unanswered question to see if that’s improved as well.

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. Incyte. May 1, 2026. Accessed May 15, 2026. https://tinyurl.com/3mpajbxx