nirogacestat nirogacestat nirogacestat (pf-03084014) is a selective gamma secretase inhibitor developed by springworks therapeutics that has potential anti-tumor activity. it was granted fda breakthrough drug designation in september 2019 for adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis. nirogacestatclinical dataother namespf-03084014atc codenoneidentifiers iupac name (s)-2-((s)-5,7-difluoro-1,2,3,4-tetrahydronaphthalen-3-ylamino)-n-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1h-imidazol-4-yl)pentanamide cas number1290543-63-3pubchem cid46224413drugbankdb12005chemspider26232306uniiqz62892ofjkeggd10960comptox dashboard (epa)dtxsid60235679 chemical and physical dataformulac27h41f2n5omolar mass489.656 g·mol−13d model (jsmol)interactive image smiles ccc(n1()ccc2=cc(f)=cc(f)=c2c1)()/c(o)=n/c3=cn(c(c)(cncc(c)(c)c)c)c=n3 inchi inchi=1s/c27h41f2n5o/c1-7-8-23(32-20-10-9-18-11-19(28)12-22(29)21(18)13-20)25(35)33-24-14-34(17-31-24)27(5,6)16-30-15-26(2,3)4/h11-12,14,17,20,23,30,32h,7-10,13,15-16h2,1-6h3,(h,33,35)/t20-,23-/m0/s1key:vfcrklwbymdaed-rewpjtcusa-n medical uses nirogacestat is currently in phase 2 clinical trials for unresectable desmoid tumors. in addition, a phase 3 clinical trial, defi, is currently in progress for nirogacestat for adults with desmoid tumors and aggressive fibromatosis. in addition, three trials are presently recruiting patients that include nirogacestat with other anticancer therapies in multiple myeloma, including the universal study for nirogacestat with the allogeneic car-t therapy allo-715.