The phase III VERIFY trial, in which phlebotomy-dependent patients with polycythemia vera were randomized to treatment with either rusfertide (PTG-300) or placebo, as an add-on to standard of care treatment, met its primary endpoint of proportion of patients achieving response.
In his first sit-down interview since beginning his role as FDA commissioner 17 days earlier, Marty Makary, a former Johns Hopkins surgeon and the only Trump pick for HHS whose confirmation received Democratic support, said he would speed up approvals for rare-disease treatments by reducing reliance on animal testing and shifting towards organoids and computational models.
