US FDA grants rusfertide Breakthrough Therapy designation for the treatment of polycythemia vera
The designation is based on data from an ongoing Phase II study, which showed that most patients treated were able to eliminate therapeutic phlebotomies and maintain a target haematocrit of <45%. It has already received orphan drug and fast track designations for this indication.
Source:
Biospace Inc.
SPS commentary:
Rusfertide (PTG-300) is an injectable hepcidin mimetic. A planned Phase III placebo-controlled study in polycythemia vera (n=200-250) will include patients who require frequent phlebotomy treatments with or without cytoreductive or other concomitant therapies.