STAT+: Moving past regulatory obstacles, Protagonist Therapeutics looks to pivotal trial for its rare blood disorder therapy
Moving past regulatory obstacles, Protagonist Therapeutics looks to pivotal trial for its rare blood disorder therapy.
FRANKFURT, Germany — Protagonist Therapeutics has run into some speed bumps as it’s developed its medicine for a rare blood cancer characterized by the overproduction of red blood cells.
In 2021, the Food and Drug Administration put studies of the drug, rusfertide, on hold after a mouse study raised concerns about skin malignancies (the hold was lifted quickly, after less than a month). Then last year, citing concerns about malignancies, the FDA yanked the therapy’s “breakthrough” designation, which can expedite a medicine’s review.
But the therapy received some primetime attention here at the European Hematology Association’s annual meeting over the weekend. In presentations at a press briefing and at a session for late-breaking studies, researchers outlined what they portrayed as promising Phase 2 results indicating rusfertide helped stabilize red blood cells at healthier levels and improved patients’ symptoms, including fatigue and brain fog. A Phase 3 trial of rusfertide is ongoing.
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