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Nirogacestat oral tablet
Nirogacestat inhibits gamma-secretase, reducing the production of Notch intracellular domain and downstream signaling that drives abnormal cell proliferation in desmoid tumors.
Nirogacestat inhibits gamma-secretase, reducing the production of Notch intracellular domain and downstream signaling that drives abnormal cell proliferation in desmoid tumors. Used for Desmoid tumors (aggressive fibromatosis).
At a glance
| Generic name | Nirogacestat oral tablet |
|---|---|
| Also known as | PF-03084014, Ogsiveo |
| Sponsor | SpringWorks Therapeutics, Inc. |
| Drug class | Gamma-secretase inhibitor |
| Target | Gamma-secretase complex (PSEN1/PSEN2) |
| Modality | Small molecule |
| Therapeutic area | Oncology |
| Phase | Phase 3 |
Mechanism of action
Gamma-secretase is an intramembrane protease complex that cleaves Notch receptors and other substrates. In desmoid tumors, aberrant Notch signaling promotes fibroblast proliferation and tumor growth. By blocking gamma-secretase activity, nirogacestat suppresses Notch-driven proliferation and induces tumor regression or stabilization. This mechanism is particularly relevant in desmoid tumors with CTNNB1 mutations that activate Wnt/β-catenin signaling, which cooperates with Notch signaling.
Approved indications
- Desmoid tumors (aggressive fibromatosis)
Common side effects
- Diarrhea
- Nausea
- Fatigue
- Vomiting
- Abdominal pain
- Decreased appetite
Key clinical trials
- A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF) (PHASE2)
- Nirogacestat in Premenopausal Females With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (PHASE4)
- A Study to Examine the Effects of Novel Therapy Linvoseltamab in Combination With Other Cancer Treatments for Adult Patients With Multiple Myeloma That is Resistant to Current Standard of Care Treatments (PHASE1)
- A Study to Assess Adverse Events and Change in Disease Activity of Intravenously (IV) Infused Etentamig (ABBV-383) in Combination With Anti-Cancer Regimens for the Treatment of Adult Participants With Relapsed/Refractory Multiple Myeloma (PHASE1)
- Nirogacestat in Ovarian Granulosa Cell Tumors (PHASE2)
- To Determine the Effect of CYP Induction Following Administration of Nirogacestat in Healthy Adult Male Participants (PHASE1)
- Evaluate the Effects of Famotidine or Rabeprazole on the PK of Nirogacestat (PHASE1)
- Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (PHASE3)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Nirogacestat oral tablet CI brief — competitive landscape report
- Nirogacestat oral tablet updates RSS · CI watch RSS
- SpringWorks Therapeutics, Inc. portfolio CI