Register today for a LIVE national broadcast on New Treatment Options for Rare Diseases in the Soft Tissue for March 5th and 6th!

News >

FDA Grants Nirogacestat Breakthrough Designation for Desmoid Tumors

Lisa Astor
Published: Wednesday, Sep 04, 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.1



  1. SpringWorks Therapeutics Receives Breakthrough Therapy Designation for Nirogacestat for the Treatment of Adult Patients with Progressive, Unresectable, Recurrent or Refractory Desmoid Tumors [press release]. Stamford, CT: SpringWorks Therapeutics, Inc; August 29, 2019. Accessed September 3, 2019.
  2. Kummar S, O’Sullivan Coyne G, Do KT, et al. Clinical Activity of the γ-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors (Aggressive Fibromatosis). J Clin Oncol. 2017;35(14):1561-1569. doi: 10.1200/JCO.2016.71.1994.

... to read the full story
To Read the Full Story

View Conference Coverage
Online CME Activities
TitleExpiration DateCME Credits
Publication Bottom Border
Border Publication