Nirogacestat, a γ-Secretase Inhibitor for Desmoid Tumors.
Mrinal GounderRavin RatanThierry AlcindorPatrick SchöffskiWinette T van der GraafBreelyn A WilkyRichard F RiedelAllison LimL Mary SmithStephanie MoodySteven AttiaSant ChawlaGina D'AmatoNoah FedermanPriscilla MerriamBrian A Van TineBruno VincenziCharlotte BensonNam Quoc BuiRashmi ChughGabriel TinocoJohn CharlsonPalma DileoLee HartnerLore LapeireFilomena MazzeoEmanuela PalmeriniPeter ReichardtSilvia StacchiottiHoward H BaileyMelissa A BurgessGregory M CoteLara E DavisHari DeshpandeHans GelderblomGiovanni GrignaniElizabeth LoggersTony PhilipJoseph G PresseyShivaani KummarBernd KasperPublished in: The New England journal of medicine (2023)
Nirogacestat was associated with significant benefits with respect to progression-free survival, objective response, pain, symptom burden, physical functioning, role functioning, and health-related quality of life in adults with progressing desmoid tumors. Adverse events with nirogacestat were frequent but mostly low grade. (Funded by SpringWorks Therapeutics; DeFi ClinicalTrials.gov number, NCT03785964.).