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FDA accepts Biologics License Application of ropeginterferon alfa-2b for polycythemia vera

Jun 14, 2020

On June 4, 2020, the U.S. Food and Drug Administration (FDA) accepted a Biologics License Application (BLA) for ropeginterferon alfa-2b (P1101) for the treatment of polycythemia vera in the absence of symptomatic splenomegaly. 1The administration’s decision is expected at the beginning of next year.

Ropeginterferon alfa-2b is an investigational treatment comprising of structurally novel mono-pegylated proline interferon, designed for administration once every two weeks. The application was supported by the positive results of the phase III PROUD/CONTI-PV study, which compared the efficacy and safety of ropeginterferon alfa-2b (n = 95) with hydroxyurea/best available therapy (HU/BAT; n = 74) in patients with polycythemia vera. 2The novel agent induced higher response rates compared with HU/BAT, with rates increasing throughout 24 months of treatment. At 36 months, 66% vs27% of patients reached a molecular response, and those responses were closely related to complete hematological response. In addition, responses were more durable with ropeginterferon alfa-2b than the comparator therapy (70.5% vs51.4% of patients maintained a complete hematological response after 36 months). The safety profile of the investigational treatment was manageable, and similar rates of adverse events (mostly anemia, thrombocytopenia, and leukopenia) were reported in both treatment groups.

Ropeginterferon alfa-2b has previously been granted FDA’s orphan drug designation for the treatment of polycythemia vera and was approved for use in Europe in 2019. 3

  1. U.S. FDA accepts PharmaEssentia’s application for ropeginterferon alfa-2b to treat polythycemia vera. http://www.pharmaessentia.com/en/news_latest. Published June 4, 2020. Accessed June 8, 2020.
  2. Gisslinger H, et al. Evidence for superior efficacy and disease modification after three years of prospective randomized controlled treatment of polycythemia vera patients with ropeginterferon alfa-2b vs. HU/BAT. Blood. 2018;132(Supplement 1):579. DOI: 1182/blood-2018-99-118715
  3. Besremi. https://www.ema.europa.eu/en/medicines/human/EPAR/besremi. Accessed June 8, 2020.