FDA Approves Ropeginterferon Alfa-2b-njft for Adults With Polcythemia Vera

The FDA has granted approval to ropeginterferon alfa-2b-njft (Besremi) for the treatment of adults with polycythemia vera (PV).¹

Ropeginterferon alfa-2b-njft is a monopegylated, long-acting interferon, which has been approved based on results from the phase 3 PROUD/CONTINUATION-PV clinical trial (NCT01949805; NCT02218047), which showed that ropeginterferon alfa-2b-njft had a high and durable hematological response in patients over 36 months, compared to hydroxyurea (Siklos).²

“The FDA approval of BESREMi for people with polycythemia vera represents the next step in advancing patient care as it provides a critical addition to managing not only symptom burden and near-term complications, but also treating the cancer early, which may help reduce the risk of disease progression over time,” said Srdan Verstovsek, MD, PhD, director of the Hanns A. Pielenz Clinical Research Center for Myeloproliferative Neoplasms, Department of Leukemia at the University of Texas MD Anderson Cancer Center, in a press release. “With the availability of an FDA-approved, next-generation interferon for this indication, it’s time that we focus on preserving the long-term health of patients with polycythemia vera.”

Three-hundred six patients were enrolled in the study and 257 patients were randomly assigned treatment, with 171 patients rolling over to the CONTINUATION-PV trial. In the PROUD-PV group, 21% of the patients in the ropeginterferon alfa-2b arm met the study endpoint of complete hematological response with normal spleen size, whereas 28% of the patients on standard therapy met this endpoint.

In the CONTINUATION-PV group, 61% of patients on ropeginterferon alfa-2b-njft met complete hematological response with improved disease burden markers. Further, 80% had a hematological response based solely on laboratory parameters.

According to previously published results, excluding the spleen size criteria, 43% of patients on ropeginterferon alfa-2b had complete hematological response compared to 46% on the standard of care group in the PROUD-PV trial. In the patients that were on the CONTINUATION-PV trial, 71% of patients on ropeginterferon alfa-2b had a complete hematological response compared to 58% on the standard of care.²

The most common grade adverse events having occurred in more than 40% of patients were influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain. Serious adverse reactions were observed in greater than 4% of patients and included were urinary tract infection, transient ischemic attack, and depression.¹

“We are incredibly proud to deliver on our goal of bringing treatments like BESREMi to the polycythemia vera community where there is clear unmet need for more effective, tolerable and durable treatments to preserve patients’ health and well-being,” said Ko-Chung Lin, PhD, co-counder and chief executive officer for PharmaEssentia and inventor of ropeginterferon alfa-2b-njft, in the press release.“As we begin working closely with the community to integrate this important treatment into clinical practice, we also continue to expand our scientific efforts to unlock the full potential of our pioneering molecule.”

_REFERENCES:

_{1. 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 November 12, 2021. https://bwnews.pr/3CdLudY}

_{2. Gisslinger H, Klade C, Georgiev P, et al. Ropeginterferon alfa-2b versus standard therapy for polycythaemia vera (PROUD-PV and CONTINUATION-PV): a randomised, non-inferiority, phase 3 trial and its extension study. Lancet Haematol. 2020;7(3):e196-e208. doi: 10.1016/S2352-3026(19)30236-4}