FDA Accepts Application for Ropeginterferon Alfa-2b for Polycythemia Vera

The FDA has accepted a Biologics License Application (BLA) for ropeginterferon alfa-2b (P1101) for use as a treatment for patients with polycythemia vera (PV) in the absence of symptomatic splenomegaly, according to PharmaEssentia Corporation, the manufacturer of the novel pegylated interferon.

A decision from the FDA on the application is expected early next year, according to the company.

"Our focus is on stunting these rare malignancies, preserving patient well-being and slowing the progression into more aggressive and deadly cancers," Meredith Manning, US General Manager for PharmaEssentia, said in a statement. "We believe ropeginterferon alfa-2b could become an important new therapeutic tool and look forward to engaging with the regulators in our efforts to introduce this option to the underserved PV community in the United States."

The BLA is based on findings from the phase 3 PROUD/CONTI-PV clinical trial, in which patients with polycythemia vera were treated with either ropeginterferon alfa-2b or hydroxyurea/best available therapy. Overall, there were 95 patients in the ropeginterferon alfa-2b arm and 74 patients in the control arm. At 36 months, the complete hematological response rate was much higher in the ropeginterferon alfa-2b group compared to the cohort receiving hydroxyurea/best available therapy. Specifically, the rates were 70.5% versus 51.4%, respectively.

The investigators observed that the response rates with ropeginterferon alfa-2b increased steadily over 24 months of therapy and stayed constant after 36 months. After 36 months of treatment, 66% of patients in the ropeginterferon alfa-2b cohort reached a molecular response compared with 27% of the patients in the cohort receiving hydroxyurea/best available therapy. According to the study researchers, the molecular responses were closely related to complete hematological responses.

Regarding the safety profile for the treatments administered in the trial, the rates of adverse events was comparable between the 2 cohorts. The most frequently reported adverse events (occurring in more than 10% of patients) considered to be related to treatment were anemia, thrombocytopenia, and leukopenia.

According to PharmaEssentia, “Ropeginterferon alfa-2b is a novel, long-acting, mono-pegylated proline interferon that has been engineered with an optimized profile to support improved pharmacokinetic properties and demonstrated tolerability and convenience compared with conventional interferons. It is designed for administration once every two weeks, or once every four weeks during long-term maintenance.”

The FDA previously granted ropeginterferon alfa-2b and Orphan Drug designation for treatment of polycythemia vera. The Orphan Drug designation is intended to expedite the development and review of treatments for rare diseases.

Ropeginterferon alfa-2b was approved by the European Commission in February 2019 for marketing in the European Union as a monotherapy for adults for the treatment of polycythemia vera without symptomatic splenomegaly.

Polycythemia vera is a rare blood cancer that causes a patient’s bone marrow to produce too many red blood cells. The additional cells thicken the patient’s blood, which can cause issues such as thrombosis and embolism. Transformation to secondary myelofibrosis or leukemia can also occur in patients.

U.S. FDA Accepts PharmaEssentia’s Application for Ropeginterferon Alfa-2b to Treat Polythycemia Vera. Published June 4, 2020. https://yhoo.it/30fZcxA. Access June 4, 2020.