Besremi Recognized by Updated Guidelines for Essential Thrombocythemia

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology now include Besremi (ropeginterferon alfa-2b-njft) as a Category 1 preferred treatment option for certain high-risk patients with essential thrombocythemia (ET) who have not responded adequately to existing therapies or whose response has diminished over time, according to a news release from PharmaEssentia.

Although Besremi is not yet approved by the U.S. Food and Drug Administration (FDA) for ET, the guideline update reflects clinical evidence, marking an important milestone for patients and clinicians navigating limited treatment options.

NCCN Update Highlights a New Option for High-Risk ET

The NCCN designation places Besremi among the most strongly recommended regimens for ET in a specific group of patients. Category 1 recommendations are based on high-level evidence and uniform consensus that the treatment is appropriate.

For patients, this update signals growing confidence in the therapy’s potential role in managing ET, particularly for those at high risk who have not benefited from standard approaches. For physicians, NCCN guideline inclusion often influences treatment planning, insurance coverage and reimbursement decisions.

ET is a rare blood cancer and a type of myeloproliferative neoplasm (MPN) in which the bone marrow produces too many platelets. Elevated platelet counts can increase the risk of serious complications, including blood clots, abnormal bleeding and spleen enlargement.

Many patients live with ET for years, but managing the disease can become challenging when current treatments stop working or cause side effects. The NCCN’s recognition of Besremi offers hope for patients who need additional options to control platelet counts and reduce long-term risks.

Understanding the Treatment: What Is Besremi?

Besremi is a long-acting interferon therapy designed to target the underlying disease process in myeloproliferative neoplasms. It is already FDA-approved for adults with polycythemia vera, another related blood cancer, and has been approved in more than 40 countries worldwide.

PharmaEssentia has submitted a supplemental biologics license application to the FDA to expand the Besremi label to include ET, and that review is currently ongoing.

How the NCCN Makes Its Recommendations

The NCCN is a nonprofit alliance of leading U.S. cancer centers that develops evidence-based guidelines used widely by oncologists and hematologists. Recommendations are informed by clinical trial data, expert consensus and real-world treatment considerations.

A Category 1 listing means experts agree, based on strong evidence, that the therapy is an appropriate option for the defined patient population. Although the NCCN update does not replace FDA approval, it often shapes real-world care decisions.

According to the updated guidelines, Besremi is recommended for patients with high-risk ET who have had an inadequate response to or lost response to other treatments. High-risk ET typically includes patients with factors such as older age or a history of blood clots, which increase the likelihood of complications.

These patients often require long-term disease control, making durable and tolerable treatment options especially important.

Additional Considerations: Safety and Monitoring

Like other interferon-based therapies, Besremi carries important safety considerations. Interferon alfa products may cause or worsen serious conditions affecting mental health, the immune system, the heart or other organs. Patients receiving the drug require regular clinical and laboratory monitoring.

Certain conditions, including severe depression, active autoimmune disease, and moderate to severe liver impairment, are contraindications. Patients are encouraged to discuss risks, monitoring requirements, and potential side effects in detail with their care team.

What This Update Means Going Forward

For patients with ET, the NCCN guideline update represents progress toward expanding evidence-based treatment options, particularly for those who have exhausted standard therapies. Although Besremi is still under FDA review for ET, its inclusion as a Category 1 preferred regimen underscores the therapy’s potential role in care.

As regulatory review continues, patients and caregivers may wish to speak with their hematologist about how evolving guidelines could influence future treatment decisions. For now, the update reflects growing momentum toward more personalized and effective management strategies for ET.

Reference

1. PharmaEssentia Announces Updated NCCN Clinical Practice Guidelines Include ropeginterferon alfa-2b-njft (BESREMi®) for Treatment of Essential Thrombocythemia (ET), by . PharmaEssentia USA Corporation. News release; Jan. 27, 2026.

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 and reviewed by a human editor.

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