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Nirogacestat oral tablet
Nirogacestat oral tablet is a Gamma-secretase inhibitor Small molecule drug developed by SpringWorks Therapeutics, Inc.. It is currently in Phase 3 development for Desmoid tumors (aggressive fibromatosis). Also known as: PF-03084014, Ogsiveo.
Nirogacestat inhibits gamma-secretase, reducing the production of Notch intracellular domain and downstream signaling that drives abnormal cell proliferation in desmoid tumors.
Nirogacestat oral tablet is a small molecule gamma-secretase inhibitor used to treat various conditions, including triple negative breast neoplasms, advanced solid tumors, and desmoid tumors. It is classified as an inhibitor and is used as a single-agent treatment in patients with advanced breast cancer and other conditions.
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Baseline phase 3 → approval rate
+58.3pp
Industry-wide phase 3 drugs reach approval ~58.3% of the time (BIO/Informa 2023 industry benchmark across all therapeutic areas). -
Oncology Phase 3 boost
+3.0pp
Oncology Phase 3 trials have higher approval rates (~61%) than the cross-industry average due to clearer endpoints and FDA oncology pathway.
| Regulator | Country | Likely year | Lag vs FDA |
|---|---|---|---|
| FDA | US | 2028–2030 | — |
| EMA | EU | 2029–2031 | +0.7 yr |
| MHRA | GB | 2029–2031 | +0.7 yr |
| Health Canada | CA | 2029–2032 | +0.9 yr |
| TGA | AU | 2029–2032 | +1.2 yr |
| PMDA | JP | 2029–2032 | +1.5 yr |
| NMPA | CN | 2030–2033 | +2.3 yr |
| MFDS | KR | 2029–2032 | +1.4 yr |
| CDSCO | IN | 2029–2033 | +1.8 yr |
| ANVISA | BR | 2030–2033 | +2.3 yr |
Hover any row for the lag rationale. Lag estimates are reduced when the drug has FDA Breakthrough or EMA PRIME designation (sponsors file globally in parallel).
Estimate based on the BIO/Informa industry phase transition rates plus per-drug modifiers for therapeutic area, sponsor type, FDA designations, mechanism, and trial design. Per-jurisdiction lags from Tufts CSDD international approval studies. Not investment, clinical or regulatory advice. Methodology: /methodology#likelihood.
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
Frequently asked questions about Nirogacestat oral tablet
What is Nirogacestat oral tablet?
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What does Nirogacestat oral tablet target?
Related
- Drug class: All Gamma-secretase inhibitor drugs
- Target: All drugs targeting Gamma-secretase complex (PSEN1/PSEN2)
- Manufacturer: SpringWorks Therapeutics, Inc. — full pipeline
- Therapeutic area: All drugs in Oncology
- Indication: Drugs for Desmoid tumors (aggressive fibromatosis)
- Also known as: PF-03084014, Ogsiveo
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing