FDA Approval of Essential Thrombocythemia Drug is “Important Milestone”

The FDA has approved ropeginterferon alfa-2b-njft (BESREMi) for adults with essential thrombocythemia, making it the second FDA-approved therapy for the disease after anagrelide (Agrylin).

Ruben Mesa, MD, FACP, president of Advocate Health’s Cancer National Service Line and executive director of the Atrium Health Wake Forest Baptist Comprehensive Cancer Center, as well as principal investigator of the phase 3 SURPASS-ET trial (NCT04285086), spoke with CancerNetwork® about what this approval means for patients with essential thrombocythemia. Describing the agent as a “major addition” to the treatment landscape, he noted that ropeginterferon has been helpful in effectively controlling blood counts among patients, which may lead to longer and better outcomes down the line.

The approval of ropeginterferon was supported by data from the SURPASS-ET trial, which showed superior durable responses and a reduction in thromboembolic events over a year of treatment with the agent vs anagrelide.

Transcript:

CancerNetwork: What does the FDA approval of ropeginterferon alfa mean for adult patients with essential thrombocythemia?

Mesa: The approval of ropeginterferon alfa for these patients is a very important milestone. Essential thrombocythemia is a chronic leukemia. In the short term, it can carry a risk of blood clots, bleeding, and difficult symptoms, and it can be a progressive disease that can progress on to myelofibrosis, acute leukemia, or, sadly, even patients [dying] if the disease were to really progress. Up to this point, we’ve only had one FDA-approved therapy: anagrelide, which is an isolated platelet-lowering therapy. Ropeginterferon is a major addition. One, it’s the second FDA-approved drug, so there have been limited options. Two, it’s been beneficial in helping to control the [blood] counts in an effective way and having an impact on the underlying clonal disease measured by the impact on the genetics of the disease. We hope this will lead to longer and better outcomes for these patients.

Reference

FDA approves PharmaEssentia’s BESREMi® (ropeginterferon alfa-2b-njft) for adults with essential thrombocythemia, a rare blood cancer. News release. PharmaEssentia USA Corporation. August 31, 2026. Accessed September 1, 2026. https://tinyurl.com/bdnt65xx