FDA Approves Application for Polythycemia Vera Treatment

PharmaEssentia Corporation, a biopharmaceutical company located in Burlington, Massachusetts, has recently seen their Biologics License Application (BLA) approved by the FDA. This application was submitted for ropeginterferon alfa-2b, a treatment of polythycemia vera. Moving forward, an agency decision is expected in the beginning of 2021.

About Polythycemia Vera

Polythycemia vera (PCV) is a rare blood cancer that is typically slow-growing. It sees the bone marrow producing too many platelets and red and white blood cells, leading to the blood becoming too thick. This causes symptoms like double vision, dizziness, headaches, itchiness, abdominal pain, excessive sweating, painful joints, weakness, shortness of breath, and abnormal blood cell counts. Complications can occur due to thickened blood, including blood clots, heart attacks, abnormal bleeding, and strokes. A mutated JAK2 gene is believed to cause these symptoms, as it regulates cell production of the bone marrow. Medical professionals do not believe that it is inherited. Doctors will treat this cancer in a variety of ways, including phlebotomy, medications that lower blood cell counts, antihistamines, and low doses of aspirin.

About Ropeginterferon Alfa-2b

This medication is a long acting mono-pegylated proline interferon that has been proven to be more tolerable and effective than conventional interferons. Medical professionals have found that it is more convenient to patients as well. It can be taken once every two weeks, which can be expanded to once every four weeks when used for long-term maintenance.

The approval of the BLA was supported by data from the Phase 3 PROUD/CONTI-PV trial. This study proved that this treatment offered symptom control and high hematologic responses while being well tolerated. Researchers concluded that ropeginterferon alfa-2b has disease-modifying capability.

Going further into this study, participants in the study were put into two groups. One was given ropeginterferon alfa-2b while the other was given the best available therapy. The former maintained a complete hematological response longer than the latter (70.5% compared to 51.4%). Those given ropeginterferon alfa-2b saw response rates rise through the first 24 months of treatment and remain steady through month 36. In fact, after these 36 months, 66% of patients experienced a molecular response. Only 27% of participants in the best available therapy group saw this response.

These results and FDA approvals are bringing hope to medical professionals and those affected by polythycemia vera.

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