Drugs / Nirogacestat
Nirogacestat
also known as
PF-03084014 · Nirogacestat dihydrobromide · Nirogacestat Hydrobromide · Ogsiveo · PF 3084014 · PF-03084014-04
Regulatory milestones approvals, filings & regulatory actions · 2 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Approved | EU (EMA) | Ogsiveo | — | 2025-08-14 | europa.eu ↗ |
| Approved | US (FDA) | OGSIVEO | — | 2023-11-27 | fda.gov ↗ |
Trials 25
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 17 trials | ||||||
| Phase 1 | NCT05556798 | Oct 2022 → Oct 2026 expected | plasma cell myeloma | Memorial Sloan Kettering Cancer Center | Active not recruiting | No outcome recorded |
| Phase 1 | NCT05137054 | Aug 2022 → Mar 2028 expected | plasma cell myeloma | Regeneron Pharmaceuticals | Recruiting | No outcome recorded |
| Phase 1 | NCT04171843 | Apr 2020 → Oct 2022 | refractory plasma cell neoplasm | Precision BioSciences, Inc. | Terminated | No outcome recorded |
| Phase 1 | NCT04093596 | Sep 2019 → Jan 2025 | refractory plasma cell neoplasm | Allogene Therapeutics | Completed | No outcome recorded |
| Phase 1 | NCT02462707 | Jul 2015 → Jun 2016 | cancer | Pfizer | Withdrawn | No outcome recorded |
| Phase 1 | NCT01876251 | Nov 2013 → Dec 2015 | breast carcinoma | Pfizer | Terminated | No outcome recorded Stop: Business |
| Phase 1 | NCT00878189 | Jun 2009 → Jan 2013 | neoplasm | Pfizer | Completed | No outcome recorded |
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT07084896 | Jun 2020 → Apr 2025 overdue | plasma cell myeloma | GlaxoSmithKline | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT04126200 | Oct 2019 → Apr 2025 overdue | plasma cell myeloma | GlaxoSmithKline | Active not recruiting | No outcome recorded |
| Phase 212 trials | ||||||
| Phase 2 | NCT07539454 | Sep 2026 → Feb 2030 expected | Kaposi's sarcoma | AIDS Malignancy Consortium | Not yet recruiting | No outcome recorded |
| Phase 2 | NCT07170644 | Aug 2025 → May 2027 expected | desmoid tumor | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Active not recruiting | No outcome recorded |
| Phase 2 | NCT05879146 | Nov 2024 → Jan 2027 expected | neoplasm | M.D. Anderson Cancer Center | Recruiting | No outcome recorded |
| Phase 2 | NCT05949099 | Aug 2023 → Feb 2028 expected | desmoid tumor | Nam Bui | Recruiting | No outcome recorded |
| Phase 2 | NCT05348356 | Aug 2022 → Jul 2025 | ovarian cancer, ovarian granulosa cell tumor | SpringWorks Therapeutics, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT05090566 | Oct 2021 → Feb 2025 | plasma cell myeloma | Pfizer | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT04195399 | Oct 2020 → Mar 2026 overdue | desmoid tumor | Children's Oncology Group | Active not recruiting | No outcome recorded |
| Phase 2 | NCT02137564 | Jul 2015 | HIV infectious disease, Kaposi's sarcoma | AIDS Malignancy Consortium | Withdrawn | No outcome recorded |
| Phase 2 | NCT02338531 | Jun 2015 → Sep 2018 | breast cancer | Jules Bordet Institute | Withdrawn | No outcome recorded |
| Phase 2 | NCT02299635 | Feb 2015 → Jan 2016 | triple-negative breast carcinoma | Pfizer | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT02109445 | Sep → Nov 2014 | malignant pancreatic neoplasm | Pfizer | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT01981551 | Oct 2013 → Dec 2018 | desmoid tumor | National Cancer Institute (NCI) | Completed | No outcome recorded |
| Phase 31 trial · 1 met primary | ||||||
| Phase 3 | NCT03785964 | Apr 2019 → Apr 2022 | desmoid tumor | SpringWorks Therapeutics, Inc. | Completed | Met primary |
| Phase 41 trial | ||||||
| Phase 4 | NCT07176689 | Sep 2025 → Feb 2031 expected | desmoid tumor | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Recruiting | No outcome recorded |
| Phase not stated2 trials | ||||||
| — | NCT02955446 | — | desmoid tumor | University of Colorado, Denver | No longer available | No outcome recorded |
| — | NCT05041036 | — | desmoid tumor | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Available | No outcome recorded |
| Comparator or background therapy6 trials | ||||||
| Phase 1 | NCT07259330 Comparator | Oct 2025 → Mar 2026 | — | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Completed | No outcome recorded |
| Phase 1 | NCT07171619 Comparator | Aug → Nov 2025 | — | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Completed | No outcome recorded |
| Phase 1 | NCT04722146 Background | Mar 2021 → Oct 2025 overdue | plasma cell myeloma | Janssen Research & Development, LLC | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT05573802 Background | Jul 2023 → Oct 2026 expected | corneal disorder, monoclonal paraproteinemia disease, plasma cell myeloma | Hellenic Society of Hematology | Recruiting | No outcome recorded |
| Phase 1/2 | NCT07150104 Background | Aug 2022 → Apr 2025 overdue | plasma cell myeloma | GlaxoSmithKline | Active not recruiting | No outcome recorded |
| Phase 1/2 | NCT07150091 Background | Jul 2022 → Apr 2025 overdue | plasma cell myeloma | GlaxoSmithKline | Active not recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 6
| Date | Issuer | Release |
|---|---|---|
| 2025-10-21 | Merck KGaA, Darmstadt, Germany | Results Merck KGaA, Darmstadt, Germany Announces Publication of Long-Term Efficacy and Safety Data from the Phase 3 DeFi Trial of OGSIVEO® (nirogacestat) in Adults with Desmoid Tumors in the Journal of Clinical Oncology emdgroup.com ↗
Long-term continuous OGSIVEO treatment for up to 4 years was associated with further tumor size reductions, increase in objective response rate, sustained improvement in desmoid tumor symptoms and consistent safety profile. |
| 2025-10-21 | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Results SpringWorks Therapeutics Announces Publication of Long-Term Efficacy and Safety Data from the Phase 3 DeFi Trial of OGSIVEO® (nirogacestat) in Adults with Desmoid Tumors in the Journal of Clinical Oncology springworkstx.com ↗
Long-term continuous OGSIVEO treatment for up to 4 years was associated with further tumor size reductions, increase in objective response rate, sustained improvement in desmoid tumor symptoms and consistent safety profile |
| 2025-08-18 | Merck KGaA, Darmstadt, Germany | Regulatory European Commission Grants Approval of Ogsiveo emdgroup.com ↗ |
| 2025-08-18 | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Regulatory European Commission Grants Approval of OGSIVEO® (nirogacestat) for the Treatment of Adults with Desmoid Tumors springworkstx.com ↗
OGSIVEO is the first and only therapy approved in the European Union (EU) to treat desmoid tumors. |
| 2025-06-20 | SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany | Regulatory SpringWorks Therapeutics Receives Positive CHMP Opinion for Nirogacestat for the Treatment of Adults with Desmoid Tumors springworkstx.com ↗ |
| 2020-12-23 | Allogene Therapeutics | Regulatory Allogene Therapeutics Receives IND Clearance from the U.S. Food and Drug Administration for ALLO-715 in Combination with Nirogacestat in Relapsed/Refractory Multiple Myeloma allogene.com ↗ |
All press releases naming this drug 12 releases
Evidence & citations 10 cited values
Every value below carries the sentence it was read from. 33 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nirogacestat | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07084896 ↗ |
| Known as | Nirogacestat dihydrobromide | ChEMBL registry synonym — accepted as the source's own label CHEMBL1770916 ↗ |
| Known as | Nirogacestat Hydrobromide | “Drug: Nirogacestat Hydrobromide” NCT04195399 ↗ |
| Known as | Ogsiveo | ChEMBL registry synonym — accepted as the source's own label CHEMBL1770916 ↗ |
| Known as | PF 3084014 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1770916 ↗ |
| Known as | PF-03084014 | “A Safety, Pharmacokinetic and Efficacy Study of a y-Secretase Inhibitor, Nirogacestat (PF-03084014), in Children and Adolescents With Progressive, Surgically Unresectable Desmoid Tumors” NCT04195399 ↗11“nirogacestat (a gamma-secretase inhibitor; PF-03084014)” PMID 33682447 ↗ Mar 2021 “PF-03084014” NCT02137564 ↗ |
| Known as | PF-03084014-04 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1770916 ↗ |
| Action | Inhibit | “gamma-secretase inhibitor” NCT00878189 ↗ |
| Modality | Small molecule | “Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage” NCT01981551 ↗ |
| Route | Oral | “PF 03084014 will be administered orally” NCT01876251 ↗ |