When ruxolitinib (Jakafi®) was approved by the U.S. Food and Drug Administration (FDA) to treat myelofibrosis (MF) ten years ago, it was celebrated as the first treatment specifically for an MPN. Three years later, it was approved to treat polycythemia vera (PV).
The landscape for patients is much different now than when ruxolitinib was initially approved. In 2019, fedratinib (Inrebic®) was approved to treat MF and earlier this month, FDA approved an interferon, specifically ropeginterferon alfa-2b-njft (BESREMi®) to treat adults with polycythemia vera. And the FDA is expected to make a decision by the end November on whether to approve pacritinib for MF patients who have severe thrombocytopenia.
In addition to these new treatment options, there is a robust pipeline of potential treatments for MF, PV and essential thrombocythemia (ET) which you can see on our clinical trials webpage. We have never been more optimistic about the potential for physicians to have an arsenal of treatment options for the MPN patients they see.
These advances would not be possible without the research on MPNs taking place around the world, including what MPNRF has funded over the past 20 years. However, there is much left to learn, including which patients are likely to progress to another MPN or acute leukemia and why. We remain committed to fund research that has the potential to have a significant impact on patients’ outcomes and quality of life.
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