FDA Grants Nirogacestat Breakthrough Designation for Desmoid Tumors

Sep 4, 2019 – The FDA has granted a breakthrough therapy designation to the investigational gamma-secretase inhibitor nirogacestat (PF-03084014) for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. The designation was based on phase I and phase II data of single-agent nirogacestat in patients with desmoid tumors. Based on the early encouraging results, the phase III DeFi trial (NCT03785964) is … (leggi tutto)