FDA OKs Ropeginterferon Alfa for Essential Thrombocytopenia

The FDA has approved ropeginterferon alfa-2b-njft (BESREMi) as a treatment for adult patients with essential thrombocytopenia, according to a press release from the developers, PharmaEssentia USA Corporation.¹ This regulatory decision represents the first approval for essential thrombocytopenia in approximately 3 decades.

Supporting data for the approval came from the international phase 3 SURPASS ET trial (NCT04285086), which demonstrated durable modified European Leukemia Net (ELN) responses and hematologic control with ropeginterferon alfa.² The trial showed superior durable response rates as well as reduced thromboembolic events over a year of treatment with the agent compared with anagrelide (Agrylin).

Safety information for ropeginterferon alfa includes warnings for a risk of serious disorders, including fatal or life-threatening neuropsychiatric, autoimmune, ischemic, and infectious disorders. The agent is contraindicated for those with a history of severe depression, suicidal ideation, or suicide attempts or hypersensitivity to interferons or any inactive ingredients.

“For nearly 30 years, people living with [essential thrombocytopenia] and the physicians treating them have had limited treatment innovation.I n my experience, patients need treatment options that not only control blood counts but also address the underlying disease,” principal trial investigator Ruben Mesa, MD, president of Advocate Health’s Cancer National Service Line, which includes Atrium Health Levine Cancer Institute and the Comprehensive Cancer Center at Atrium Health Wake Forest Baptist, stated in the press release.¹ “The approval of [ropeginterferon alfa] provides an important new treatment option that is supported by strong clinical evidence and that also works at the source of the disease rather than solely managing symptoms.”

The FDA previously accepted the supplemental biologics license application for ropeginterferon alfa in January 2026.³ Additionally, ropeginterferon alfa earned FDA approval as a treatment for polycythemia vera in November 2021.⁴

References

1. 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 August 31, 2026. https://tinyurl.com/bdnt65xx
2. Ropeginterferon Alfa-2b (P1101) vs. anagrelide in essential thrombocythemia patients with hydroxyurea resistance or intolerance (SURPASS ET). ClinicalTrials.gov. Updated July 22, 2026. Accessed August 31, 2026. https://tinyurl.com/2wnpews4
3. FDA confirms a PDUFA goal date of August 30, 2026 for the sBLA submission of ropeginterferon alfa-2b-njft in essential thrombocythemia (ET). News release. PharmaEssentia USA Corporation. January 13, 2026. Accessed August 31, 2026. https://tinyurl.com/4j7y8an6
4. U.S. FDA approves BESREMi (ropeginterferon alfa-2b-njft) as the only interferon for adults with polycythemia vera. News release. PharmaEssentia Corporation. November 12, 2021. Accessed August 31, 2026. https://bwnews.pr/2YHruCT