Drug Type Small molecule drug |
Synonyms PF 3084014, PF-03084014, PF-03084014-04 + [2] |
Target |
Action inhibitors |
Mechanism γ-secretase inhibitors(Gamma-secretase inhibitors) |
Therapeutic Areas |
Active Indication |
Inactive Indication |
Originator Organization |
Active Organization |
Inactive Organization |
License Organization |
Drug Highest PhaseApproved |
First Approval Date United States (27 Nov 2023), |
RegulationPriority Review (United States), Breakthrough Therapy (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union) |
Molecular FormulaC27H43Br2F2N5O |
InChIKeyLXEYYZYDWLAIPW-KBVFCZPLSA-N |
CAS Registry1962925-29-6 |
Indication | Country/Location | Organization | Date |
---|---|---|---|
Aggressive Fibromatosis | United States | 27 Nov 2023 |
Indication | Highest Phase | Country/Location | Organization | Date |
---|---|---|---|---|
Corneal Diseases | Phase 2 | Greece | 14 Jul 2023 | |
Granulosa Cell Tumor of the Ovary | Phase 2 | United States | 30 Aug 2022 | |
Granulosa Cell Tumor of the Ovary | Phase 2 | Canada | 30 Aug 2022 | |
Granulosa Cell Tumor of the Ovary | Phase 2 | Poland | 30 Aug 2022 | |
Multiple Myeloma | Phase 2 | United States | 08 Jun 2020 | |
Multiple Myeloma | Phase 2 | Australia | 08 Jun 2020 | |
Multiple Myeloma | Phase 2 | Canada | 08 Jun 2020 | |
Multiple Myeloma | Phase 2 | France | 08 Jun 2020 | |
Multiple Myeloma | Phase 2 | Germany | 08 Jun 2020 | |
Multiple Myeloma | Phase 2 | Greece | 08 Jun 2020 |
Not Applicable | Aggressive Fibromatosis familial adenomatous polyposis (FAP) | 13 | kuarctrubz(jvwfpvdjas) = xbszjadoji snkrxqqydf (tnkvidmtak ) View more | Positive | 21 Mar 2025 | ||
Phase 3 | 70 | czzpomxcer(ipblgnxpaq) = fcvemvdecm yynqzmmueg (ydqicusogw ) View more | Positive | 20 Mar 2025 | |||
Phase 3 | 142 | byfdhbllqv(ougwstdmyf) = diarrhea was the most frequently reported adverse event xrxuhjteoz (iqlhrakxnj ) View more | Positive | 23 Jan 2025 | |||
Placebo | |||||||
Phase 3 | 142 | Nirogacestat 150 mg | khdwbcqieq(vjjukltmxs) = unmbxjkugs bopkcvdmee (fsgjsdumsr ) View more | Positive | 07 Nov 2024 | ||
Placebo | - | ||||||
Phase 3 | Aggressive Fibromatosis APC Mutation | 29 | hdjkcmaopy(nvkvcyeuxz) = jwwmkocphq bmrriqkuyu (lhfidjpdyh ) View more | Positive | 24 May 2024 | ||
Placebo | hdjkcmaopy(nvkvcyeuxz) = hvweuwbmdc bmrriqkuyu (lhfidjpdyh ) View more | ||||||
Phase 3 | Aggressive Fibromatosis CTNNB1 | 142 | fszvjalxzf(okrbujuhmk) = yremnmnbxe lebvkopkya (sdnkrcokgw, 0.11 - 0.70) View more | Positive | 24 May 2024 | ||
fszvjalxzf(okrbujuhmk) = rlbvvefrsb lebvkopkya (sdnkrcokgw, 0.13 - 0.80) View more | |||||||
Phase 3 | 144 | Nirogacestat (niro) 150 mg | rgsmpyizwd(vygrvwitta) = mzjmdgipxn uibnvnadqc (srvsskveqc ) | Positive | 15 Mar 2024 | ||
Placebo | rgsmpyizwd(vygrvwitta) = vaqpcfnrba uibnvnadqc (srvsskveqc ) | ||||||
NCT03785964 (DeFi, FDA) Manual | Phase 3 | 142 | znvxnffnur(spbgjqwfox) = njbzimggdy egsatubmdn (czybjejgpb ) View more | Positive | 27 Nov 2023 | ||
Placebo | znvxnffnur(spbgjqwfox) = vbchyzyjng egsatubmdn (czybjejgpb, 8.4 - NR) View more | ||||||
Phase 3 | 142 | fcxsjjwqkp(aoypckmtzn) = Nirogacestat significantly improved mean physical functioning score from baseline per the GODDESS DTIS PF domain compared with placebo at the pre-specified time point. The GODDESS DTIS PF domain captures varying degrees of vigorous and moderate daily activity, including moving and reaching. fhixkdndyz (fojfiqsxup ) View more | Positive | 01 Nov 2023 | |||
Placebo | |||||||
Phase 3 | 122 | knykcoxizy(uejhwmaoyg) = kzpfgeudor icrzzugopa (ksvdcjumnf, –100.0 - 517.2) View more | Positive | 31 May 2023 | |||
Placebo | knykcoxizy(uejhwmaoyg) = ecrqpbtseg icrzzugopa (ksvdcjumnf, –88.6 - 441.7) View more |