nirogacestat
Trade name: Ogsiveo
Small moleculeapprovedOrphan Drug FDAFast Track FDABreakthrough Therapy FDA
Approved
Nov 27, 2023
Clinical trial activity
28 trials · 20 clinical orgs · 1 marketing orgs
Phase 1
13
Phase 2
17
Phase 3
1
Phase 4
1
Earliest trial started Jun 1, 2009 (NCT00878189)
Timeline
2000s
- Jan 1, 2009
Earliest Phase 1 Sponsor(trial)
2010s
- Jan 1, 2013
Earliest Phase 2 Sponsor(trial)
- Jun 7, 2018
Orphan Drug Designation
- Jan 1, 2019
SpringWorks Therapeutics, Inc. — Earliest Phase 3 Sponsor(trial)
2020s
- Nov 27, 2023
SpringWorks Therapeutics, Inc. — Earliest FDA Approval
- Nov 27, 2023
SpringWorks Therapeutics, Inc. — Marketing Organization
- Nov 27, 2023
SpringWorks Therapeutics, Inc. — NDA Organization
Indications
Approved for
Studied for
- Blood Protein Disorders · Phase 1/Phase 2
- Breast Neoplasms · Phase 2
- Corneal Diseases · Phase 1/Phase 2
- Desmoid Tumors · Phase 4
- Healthy Volunteers · Phase 1
- Hematology · Phase 1/Phase 2
- HIV Infections · Phase 2
- Multiple Myeloma · Phase 2
- Neoplasms · Phase 4
- Neoplasms by Histologic Type · Phase 1
- Neoplasms, Plasma Cell · Phase 1/Phase 2
- Ovarian Neoplasms · Phase 2
- Pancreatic Neoplasms · Phase 2
- Paraproteinemias · Phase 1/Phase 2
- Sarcoma, Kaposi · Phase 2
- Triple Negative Breast Neoplasms · Phase 2
Mechanism of action
- Presenilin 1INHIBITOR
PSEN1 inhibitor
- Gamma-secretaseINHIBITOR
Gamma-secretase inhibitor
Approval history
- approvedPriority reviewFast trackBreakthroughNov 27, 2023
Chemistry & pharmacology
Loading structure…
SMILES
CCC[C@H](N[C@H]1CCc2cc(F)cc(F)c2C1)C(=O)Nc1cn(C(C)(C)CNCC(C)(C)C)cn1- Mol. weight
- 489.66 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- No
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
First in class
Sources
- ChEMBLCHEMBL1770916 ↗
- ChEMBLCHEMBL4298153 ↗
Also known as
- 2-(5,7-difluoro-1,2,3,4-tetrahydronaphthalen-3-ylamino)-n-(1-(2-methyl-1-(neopentylamino)propan-2-yl)-1h-imidazol-4-yl)pentanamide
- nirogacestat
- pf03084014
- pf 03084014
- pf-03084014
- pf-03084014
- pf-03084014
- pf3084014
- pf 3084014
- pf-3084014
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07259330 | Phase 1 | Nov 17, 2025 | ICON, SpringWorks Therapeutics, Inc. |
| NCT07176689 | Phase 4 | Sep 17, 2025 | Netherlands Cancer Institute, SpringWorks Therapeutics, Inc. |
| NCT07171619 | Phase 1 | Sep 9, 2025 | Medpace, Inc., SpringWorks Therapeutics, Inc. |
| NCT07170644 | Phase 2 | Aug 8, 2025 | SpringWorks Therapeutics, Inc. |
| NCT05879146 | Phase 2 | Oct 30, 2024 | SpringWorks Therapeutics, Inc., University of Texas at Houston |
| NCT05949099 | Phase 2 | Aug 15, 2023 | SpringWorks Therapeutics, Inc., Stanford University |
| NCT05573802 | Phase 1/Phase 2 (Phase 2) | Oct 24, 2022 | GlaxoSmithKline, Hellenic Society of Hematology, National Kapodistrian University of Athens |
| NCT05259839 | Phase 1 | Oct 20, 2022 | Teneobio, Inc. |
| NCT05556798 | Phase 1 | Oct 4, 2022 | Memorial Sloan-Kettering Cancer Center |
| NCT05348356 | Phase 2 | Sep 27, 2022 | SpringWorks Therapeutics, Inc. |
| NCT07150104 | Phase 1/Phase 2 (Phase 2) | Aug 31, 2022 | GlaxoSmithKline |
| NCT07150091 | Phase 1/Phase 2 (Phase 2) | Jul 19, 2022 | GlaxoSmithKline |
| NCT05090566 | Phase 1/Phase 2 (Phase 2) | Oct 28, 2021 | Pfizer |
| NCT04722146 | Phase 1 | Mar 12, 2021 | Janssen Pharmaceutica NV, Johnson and Johnson |
| NCT07084896 | Phase 1/Phase 2 (Phase 2) | Jun 8, 2020 | GlaxoSmithKline |
| NCT04195399 | Phase 2 | Mar 11, 2020 | National Cancer Institute (NCI), SpringWorks Therapeutics, Inc. |
| NCT04126200 | Phase 2 | Oct 7, 2019 | GlaxoSmithKline |
| NCT03785964 | Phase 3 | May 15, 2019 | SpringWorks Therapeutics, Inc. |
| NCT02955446 | N/A | Nov 2, 2016 | Pfizer, University of Colorado, Denver |
| NCT02137564 | Phase 2 | Jul 1, 2015 | AIDS Malignancy Consortium, National Cancer Institute (NCI), The EMMES Corporation |
Showing 20 of 28 trials
Page 1 / 2
Organizations
Research & Development (20)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| SpringWorks Therapeutics, Inc. | For profit | 10 | 7 | 5 | 2019 |
| Pfizer | For profit | 7 | 6 | 3 | 2009 |
| GlaxoSmithKline | For profit | 5 | 4 | 1 | 2019 |
| National Cancer Institute (NCI) | Government | 3 | 2 | 1 | 2013 |
| Academic GI Cancer Consortium | Academic/Hospital | 1 | 0 | 1 | 2014 |
| AIDS Malignancy Consortium | Academic/Hospital | 1 | 1 | 1 | 2015 |
| Free University of Brussels | Academic/Hospital | 1 | 1 | 1 | 2015 |
| Hellenic Society of Hematology | Academic/Hospital | 1 | 1 | 1 | 2022 |
| ICON | For profit | 1 | 0 | 1 | 2025 |
| Janssen Pharmaceutica NV | For profit | 1 | 1 | 1 | 2021 |
| Johnson and Johnson | For profit | 1 | 0 | 1 | 2021 |
| Medpace, Inc. | For profit | 1 | 0 | 1 | 2025 |
| Memorial Sloan-Kettering Cancer Center | Academic/Hospital | 1 | 1 | 1 | 2022 |
| National Kapodistrian University of Athens | Academic/Hospital | 1 | 0 | 1 | 2022 |
| Netherlands Cancer Institute | Academic/Hospital | 1 | 0 | 1 | 2025 |
| Stanford University | Academic/Hospital | 1 | 1 | 1 | 2023 |
| Teneobio, Inc. | For profit | 1 | 1 | 1 | 2022 |
| The EMMES Corporation | For profit | 1 | 0 | 1 | 2015 |
| University of Colorado, Denver | Academic/Hospital | 1 | 1 | 1 | 2016 |
| University of Texas at Houston | Academic/Hospital | 1 | 1 | 1 | 2024 |
20 organizations
Page 1 / 1
Marketing (2)
| Organization | Org type | Relationship | Date |
|---|---|---|---|
| SpringWorks Therapeutics, Inc. | For profit | MKTG | Nov 27, 2023 |
| SpringWorks Therapeutics, Inc. | For profit | NDA | Nov 27, 2023 |