Incyte Secures FDA Approval for Jakafi XR™ (ruxolitinib) Extended-Release Tablets Across Multiple Hematologic Indications
Incyte has announced that the U.S. Food and Drug Administration (FDA) has approved Jakafi XR™ (ruxolitinib) extended-release tablets, introducing a new once-daily treatment option for patients across several hematologic conditions. The approval marks an important milestone in expanding therapeutic flexibility for individuals living with myeloproliferative neoplasms (MPNs) and graft-versus-host disease (GVHD), while building on the established clinical role of ruxolitinib.
Broad Indications Across Hematologic Diseases
The newly approved extended-release formulation is indicated for multiple patient populations. These include adults with intermediate- or high-risk myelofibrosis (MF), a serious bone marrow disorder that disrupts normal blood cell production. It is also approved for adults with polycythemia vera (PV) who have demonstrated an inadequate response to or intolerance of hydroxyurea, a commonly used first-line therapy.
In addition, Jakafi XR is now available for adults and pediatric patients aged 12 years and older with steroid-refractory acute GVHD or chronic GVHD after failure of one or two lines of systemic therapy. GVHD is a potentially life-threatening complication that can occur following allogeneic stem cell transplantation, where donor immune cells attack the recipient’s tissues.
By covering these multiple indications, Jakafi XR extends the reach of ruxolitinib, a Janus kinase (JAK) inhibitor that has played a transformative role in hematology since its initial approval more than a decade ago.
Advancing Convenience with Once-Daily Dosing
One of the key advantages of Jakafi XR lies in its extended-release formulation, which allows for once-daily (QD) dosing. This represents a meaningful improvement in convenience compared to the immediate-release (IR) version of ruxolitinib, which is typically administered twice daily (BID).
For patients managing chronic and often complex conditions like MF, PV, or GVHD, reducing dosing frequency can simplify treatment regimens and potentially improve adherence. Many patients in these populations are already dealing with multiple medications and comorbidities, making streamlined dosing schedules particularly valuable.
Bill Meury, Chief Executive Officer of Incyte, emphasized that the approval reinforces the company’s leadership in hematology and its commitment to addressing evolving patient needs. He noted that Jakafi XR offers physicians and patients an additional option without altering the well-established therapeutic role of Jakafi in clinical practice.
Clinical Evidence Supporting Approval
The FDA’s decision was based on pharmacokinetic data demonstrating that Jakafi XR provides drug exposure comparable to the immediate-release formulation. Specifically, a single 55 mg extended-release tablet taken once daily was shown to be bioequivalent to a 25 mg immediate-release tablet taken twice daily.
Bioequivalence means that the extended-release version delivers similar levels of the active ingredient in the bloodstream over time, maintaining consistent therapeutic exposure throughout the day. This finding supports the expectation that Jakafi XR will offer similar clinical benefits to the original formulation while enhancing dosing convenience.
Although the approval was not based on new efficacy trials, the extensive clinical experience with ruxolitinib across its approved indications provides a strong foundation for its safety and effectiveness.
Established Safety Profile
The safety profile of Jakafi XR is consistent with that of the immediate-release formulation, which has been evaluated in multiple well-controlled clinical studies involving patients with MF, PV, and GVHD.
Common adverse reactions vary depending on the specific condition being treated. In patients with myelofibrosis and polycythemia vera, frequently reported side effects include thrombocytopenia (low platelet counts), anemia (low red blood cell counts), bruising, dizziness, headache, and diarrhea.
Among patients with acute GVHD, common adverse events include low platelet counts, reduced red and white blood cell counts, infections, and fluid retention or swelling. In chronic GVHD, patients may experience anemia, thrombocytopenia, and infections, including viral infections.
These safety considerations underscore the importance of ongoing monitoring and individualized patient management when using ruxolitinib-based therapies.
Clinical Perspective on Treatment Impact
Naveen Pemmaraju, M.D., Professor of Leukemia at MD Anderson Cancer Center, highlighted the significance of this approval from a clinical standpoint. He noted that patients with chronic hematologic conditions often face challenges in managing complex treatment regimens, particularly when multiple therapies are involved.
Since its original approval in 2011, ruxolitinib has significantly changed the treatment landscape for patients with MPNs and GVHD, offering improved symptom control and disease management. The availability of a once-daily extended-release option provides an additional level of flexibility, enabling physicians to tailor treatment plans to individual patient needs.
Researchers at MD Anderson have been actively involved in the clinical development of ruxolitinib, contributing to the growing body of evidence supporting its use across various hematologic conditions.
Supporting Patient Access and Education
In addition to advancing its therapeutic portfolio, Incyte continues to focus on improving patient access to its medicines. Eligible patients in the United States who are prescribed Jakafi XR can benefit from the company’s IncyteCARES program (Connecting to Access, Reimbursement, Education and Support).
This comprehensive support initiative is designed to help patients navigate the complexities of treatment, including financial assistance, insurance coverage, and educational resources. By addressing potential barriers to access, the program aims to ensure that patients can receive and adhere to prescribed therapies more effectively.
The approval of Jakafi XR represents a natural evolution in the development of ruxolitinib, building on its established efficacy and safety profile while introducing a more convenient dosing option. As treatment paradigms continue to evolve, innovations like extended-release formulations play an important role in enhancing patient experience and optimizing long-term disease management.
For patients living with myelofibrosis, polycythemia vera, and graft-versus-host disease, the availability of Jakafi XR offers a new level of flexibility without compromising therapeutic effectiveness. For clinicians, it provides an additional tool to tailor treatment strategies in a way that aligns with patient preferences and clinical needs.
Ultimately, this approval underscores Incyte’s ongoing commitment to advancing hematologic care and improving outcomes for patients facing complex and chronic conditions.
About Myelofibrosis
Myelofibrosis (MF) is a part of a group of rare, chronic blood cancers called myeloproliferative neoplasms, or MPNs. MF occurs when a bone marrow defect results in an abnormal production of blood cells, causing scar tissue to form, which can lead to severe anemia, weakness, fatigue and an enlarged spleen and liver. MF can result from a progression of other bone marrow diseases, including polycythemia vera and essential thrombocythemia, or it can occur on its own.3
About Polycythemia Vera
Polycythemia Vera (PV) is a part of a group of rare, chronic blood cancers called myeloproliferative neoplasms, or MPNs.3 PV occurs when bone marrow produces too many red blood cells, white blood cells and often platelets. Increased red blood cells and platelets can cause the blood to thicken, leading to an increased risk of blood clotting complications, including deep vein thrombosis, stroke or heart attack.4
About Graft-Versus-Host Disease
Graft-versus-host disease (GVHD) is a condition that can occur after an allogeneic stem cell transplant (the transfer of stem cells from a donor) in which the donated cells initiate an immune response and attack the transplant recipient’s organs, leading to significant morbidity and mortality. There are two major forms of GVHD: acute, which generally occurs within 100 days of transplant, and chronic, which generally occurs after 100 days of transplant. Both forms can affect multiple organ systems, including the skin, gastrointestinal (digestive) tract and liver.5
About Jakafi XR™ (ruxolitinib) Extended-Release Tablets
Jakafi XR™ (ruxolitinib) extended-release tablets are a once-daily (QD) formulation of ruxolitinib, a first-in-class JAK1/JAK2 inhibitor, approved by the U.S. FDA for the treatment of intermediate- or high-risk myelofibrosis, including primary myelofibrosis, post-polycythemia vera myelofibrosis, and post-essential thrombocythemia myelofibrosis in adults; polycythemia vera in adults who have had an inadequate response to or are intolerant of hydroxyurea; steroid-refractory acute graft-versus-host disease in adult and pediatric patients 12 years and older; and chronic graft-versus-host disease after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.
It is not known if Jakafi XR is safe or effective in children for treatment of myelofibrosis or polycythemia vera. Jakafi XR and the Jakafi XR logo are trademarks of Incyte.
About Jakafi® (ruxolitinib)
Jakafi® (ruxolitinib) is a JAK1/JAK2 inhibitor approved by the U.S. FDA for the treatment of polycythemia vera (PV) in adults who have had an inadequate response to or are intolerant of hydroxyurea; intermediate or high-risk myelofibrosis (MF), including primary MF, post-polycythemia vera MF and post-essential thrombocythemia MF in adults; steroid-refractory acute GVHD in adult and pediatric patients 12 years and older; and chronic GVHD after failure of one or two lines of systemic therapy in adult and pediatric patients 12 years and older.
It is not known if Jakafi is safe or effective in children for treatment of myelofibrosis or polycythemia vera.
Jakafi is marketed by Incyte in the U.S. and by Novartis as Jakavi® (ruxolitinib) outside the U.S. Jakafi and the Jakafi logo are registered trademarks of Incyte. Jakavi is a registered trademark of Novartis AG in countries outside the United States.
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