FDA approval of ropeginterferon alfa-2b-njft for the treatment of adult patients with polycythemia vera

On November 12, 2021, it was announced that the U.S. Food and Drug Administration (FDA) approved ropeginterferon alfa-2b-njft, a monopegylated, long-acting interferon, for the treatment of adult patients with polycythemia vera. The decision was based on results from the PEGINVERA (NCT01193699) and PROUD/CONTINUATION-PV studies (NCT01949805 and NCT02218047, respectively).¹

After 7.5 years of treatment, complete hematologic response, which was defined as a hematocrit of <45% without phlebotomy for at least 2 months, a platelet count ≤400 × 10⁹/L, a leukocyte count ≤10 × 10⁹/L, and a normal spleen size (longitudinal diameter ≤12 cm for females and ≤13 cm for males), was achieved in 61% of patients in the PEGINVERA trial. Also, based on objective laboratory parameters only, (with the exclusion of normal spleen size and thrombosis), a hematologic response was achieved in 80% of patients.¹

Pooled safety data from the trials showed serious adverse reactions with an incidence >4% included urinary tract infections, transient ischemic attacks, and depression; while the most common adverse reactions, with an incidence >40%, included influenza-like illness, arthralgia, fatigue, pruritis, nasopharyngitis, and musculoskeletal pain.¹

The recommended dose of ropeginterferon alfa-2b-njft is once every 2 weeks until hematologic parameters are stabilized. After a year, those with a stable complete hematologic response can be treated once every 4 weeks. This allows a flexible dosing that can help meet the individual needs of a patient.¹

Ropeginterferon alfa-2b-njft has previously received orphan drug designation by the FDA and is already approved in Europe, Taiwan, and South Korea for this indication.¹

For more information on the 5-year results from the PROUD-PV and CONTINUATION-PV studies read our summary article here.