Less than a year after granting breakthrough therapy designation to Protagonist Therapeutics’ treatment for a rare blood cancer, the U.S. FDA wants to rescind its decision.
Back in June of 2021, Protagonist's investigational new drug candidate rusfertide received breakthrough designation for treating patients with polycythemia vera, a rare blood cancer that can cause the bone marrow to make too many red blood cells, thickening the blood which can lead to serious problems like blood clots.
Breakthrough therapy designation, designed for drugs that treat a serious or life-threatening conditions, helps expedite clinical trials and manufacturing development strategy to get drugs to consumers faster.
But now, the FDA has sent a letter to the Newark, California-based biotech, saying it wants to revoke rusfertide’s status as a breakthrough therapy. In response, Protagonist is asking the FDA for a meeting to make their case. They also sent a brief to the agency in an attempt to keep the drug from losing its status.
The FDA has said its letter does not relate to rusfertide’s current Fast Track Designation which remains active as well as its orphan drug status. Protagonist will also stick to its original development plan and timeline, with the phase 3 study currently underway.