> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/nirogacestat-3fcb771a-72a0-450b-adde-a88a42aee6e0
> Record changed: 2026-10-13

# Nirogacestat

Highest phase reached

Approved

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

Drug type

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

First approval year

2023

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

## Development status

**First approval year**

2023

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**Listing at source**

Listed

**Highest phase reached**

Approved

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

## Names and chemistry

**Drug type**

Small molecule

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**Drug name**

Nirogacestat

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**Molecular weight**

489.66

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**Structure (SMILES)**

CCC\[C@H\](N\[C@H\]1CCc2cc(F)cc(F)c2C1)C(=O)Nc1cn(C(C)(C)CNCC(C)(C)C)cn1

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

### Other names (19)

- elranatamab nirogacestat
- nirogacestat
- Nirogacestat
- Nirogacestat (Gamma Secretase Inhibitor)
- nirogacestat \[INN\]
- Nirogacestat \[MI\]
- NIROGACESTAT \[USAN\]
- Nirogacestat \[WHO-DD\]
- nirogacestat inn
- nirogacestat mi
- Nirogacestat oral tablet
- nirogacestat usan
- nirogacestat who dd
- pf 03084014
- PF 03084014
- pf 3084014
- PF 3084014
- PF-03084014
- PF-3084014

## Identifiers

**ATC classification codes**

- L01XX81

Sources for recorded values

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**CAS registry numbers**

- 1290543-63-3

**ChEMBL ID**

CHEMBL1770916

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**InChIKey**

VFCRKLWBYMDAED-REWPJTCUSA-N

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL1770916)

**PubChem compound IDs**

- 46224413

**RxNorm IDs (RxCUI)**

- 2670631

**FDA ingredient codes (UNII)**

- QZ62892OFJ

## Regulatory information

### United States

**Applicant**

SPRINGWORKS

**Approval date**

27 Nov 2023

**Application number**

NDA217677

### European Union

**Status**

Authorised

**Status date**

14 Aug 2025

**Authorisation date**

14 Aug 2025

**Approved indications**

Ogsiveo as monotherapy is indicated for the treatment of adult patients with progressing desmoid tumours who require systemic treatment.

**Product number**

EMEA/H/C/006071

**Authorisation holder**

Merck Europe B.V.

**Priority medicines scheme**

No

**Generic**

No

**Biosimilar**

No

**Generic medicines**

0

**Biosimilar medicines**

0

**Advanced therapy**

No

**Conditional approval**

No

**Additional monitoring**

Yes

**Accelerated assessment**

No

**Exceptional circumstances**

No

#### Therapeutic areas

- Desmoid Tumors

European medicines record

Source: [www.ema.europa.eu](https://www.ema.europa.eu/en/medicines/human/EPAR/ogsiveo)

### Other regulatory facts

**Patents**

0

**Designations**

6

**Earliest recorded loss of exclusivity**

18 Aug 2035

**First in class**

Yes

**Fast track**

Yes

**Breakthrough designation**

Yes

**Orphan designation**

Yes

**Priority review**

Yes

**Orphan exclusivity**

No

**Accelerated approval**

No

**Availability**

Prescription only

## Pharmacology

### Drug classification

**Pharmacology name**

nirogacestat

**Type**

Synthetic organic

**Drug name stem**

\-stat

**Approval recorded by Guide to Pharmacology**

Yes

#### Administration routes

- Oral

### Mechanisms of action (6)

- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
P49768
- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
P49810
- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
Q8WW43
- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
Q92542
- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
Q96BI3
- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

**Protein identifier**  
Q9NZ42

### Open Targets mechanisms

- **Mechanism**  
Gamma-secretase inhibitor

**Action type**  
Inhibitor

**Target**  
Gamma-secretase

### Investigated indications (7)

- **Condition**  
Fibroma

**Highest recorded phase**  
2

**Condition identifier**  
EFO:0000497

**Subject identifier**  
D005350
- **Condition**  
Neoplasms

**Highest recorded phase**  
3

**Condition identifier**  
EFO:0000616

**Subject identifier**  
D009369
- **Condition**  
Multiple Myeloma

**Highest recorded phase**  
2

**Condition identifier**  
EFO:0001378

**Subject identifier**  
D009101
- **Condition**  
Pancreatic Neoplasms

**Highest recorded phase**  
2

**Condition identifier**  
EFO:0002618

**Subject identifier**  
D010190
- **Condition**  
Breast Neoplasms

**Highest recorded phase**  
2

**Condition identifier**  
EFO:0003869

**Subject identifier**  
D001943
- **Condition**  
Desmoid Tumors

**Highest recorded phase**  
3

**Condition identifier**  
EFO:0009907

**Subject identifier**  
D018222
- **Condition**  
Breast Neoplasms

**Highest recorded phase**  
2

**Condition identifier**  
MONDO:0007254

**Subject identifier**  
D001943

### Open Targets indications (15)

- **Condition identifier**  
EFO\_0000616
- **Condition identifier**  
EFO\_0001378

**Highest recorded phase**  
2
- **Condition identifier**  
EFO\_0003869

**Highest recorded phase**  
2
- **Condition identifier**  
EFO\_0009907
- **Condition identifier**  
MONDO\_0004963

**Highest recorded phase**  
1
- **Condition identifier**  
MONDO\_0004975

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0005031

**Highest recorded phase**  
3
- **Condition identifier**  
MONDO\_0005055

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0005070

**Highest recorded phase**  
4
- **Condition identifier**  
MONDO\_0005184

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0005494

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0007254

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0007608

**Highest recorded phase**  
4
- **Condition identifier**  
MONDO\_0009693

**Highest recorded phase**  
2
- **Condition identifier**  
MONDO\_0023283

**Highest recorded phase**  
2

### Reported adverse events

- **Event**  
ureteric haemorrhage

**Reports**  
2

**Reporting signal score**

20.0931

### Trial references (19)

- NCT00878189  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT00878189)
- NCT02462707  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02462707)
- NCT05879146  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05879146)
- NCT01876251  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01876251)
- NCT02338531  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02338531)
- NCT01981551  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT01981551)
- NCT03785964  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03785964)
- NCT04195399  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04195399)
- NCT05949099  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05949099)
- NCT02109445  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02109445)
- NCT02299635  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02299635)
- NCT04093596  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04093596)
- NCT04126200  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04126200)
- NCT04171843  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04171843)
- NCT04722146  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04722146)
- NCT05090566  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05090566)
- NCT05137054  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05137054)
- NCT05259839  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05259839)
- NCT05556798  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT05556798)

## Dosing

**Dose**

150 mg

**Route**

Oral

**How often**

Twice daily

### Conditions of use (2)

- Swallow tablets whole, do not break, crush, or chew
- Take with or without food

## Chemical properties

**Hydrogen bond acceptors**

5

**Hydrogen bond donors**

3

**Polar surface area**

70.98

**Calculated partition coefficient (ALogP)**

4.79

**Calculated partition coefficient (XLogP)**

4.8

**Prodrug**

No

**Chirality**

Single enantiomer

**Inorganic**

No

**Rule-of-five violations**

0

**Rotatable bonds**

10

**Heavy atoms**

35

**Molecular formula**

C27H41F2N5O

**Monoisotopic mass**

489.32791727

**Natural product**

No

## Evidence counts

**Recorded assays**

366

**Active assays**

71

### Active assay types

- Confirmatory

## Pharmacologic actions

- Gamma Secretase Inhibitors and Modulators  
Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D000091062.html)
- Antineoplastic Agents  
Source: [id.nlm.nih.gov](https://id.nlm.nih.gov/mesh/D000970.html)

## Biological measurements

### Observed measurements

#### Oral bioavailability

**Median**

19

**Mean**

19

**Minimum**

19

**Maximum**

19

**Unit**

%

**Assays**

1

**Standard deviation**

0

**Summary method**

median

**Censored results excluded**

0

**Other units excluded**

0

**Unknown units excluded**

0

#### Measurement types

- F

## Related records

### Trials (45)

- A Dose-escalation Study to Evaluate the Safety and Clinical Activity of PBCAR269A, With or Without Nirogacestat, in Study Participants With Relapsed/Refractory Multiple Myeloma
- A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations
- A Study Evaluating The PF-03084014 In Combination With Docetaxel In Patients With Advanced Breast Cancer
- A Study of Belantamab Mafodotin in Combination With Nirogacestat and Pomalidomide in People With Multiple Myeloma That Has Not Responded to Treatment or Has Come Back After Treatment
- A Study of Nirogacestat in Japanese Adults With Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
- A Study Of PF-03084014 In Japanese Patients With Advanced Solid Tumors
- A Study of RO4929097 in Patients With Advanced Renal Cell Carcinoma That Have Failed Vascular Endothelial Growth Factor (VEGF)/Vascular Endothelial Growth Factor Receptor (VEGFR) Therapy
- A Study of Teclistamab With Other Anticancer Therapies in Participants With Multiple Myeloma
- [A Study to Examine the Effects of Novel Therapy Linvoseltamab in Combination With Other Cancer Treatments for Adult Participants With Multiple Myeloma That is Resistant to Current Standard of Care Treatments](https://trialight.com/data/trial/a-study-to-examine-the-effects-of-novel-therapy-4622f07a-ab31-4a56-a46c-08b2e34a9258.md)
- A Study to Investigate Safety and Clinical Activity of Belantamab Mafodotin in Combination With Lenalidomide, Dexamethasone and Nirogacestat in Patients With Transplant Ineligible Newly Diagnosed Multiple Myeloma
- A Trial In Patients With Advanced Cancer And Leukemia
- BCMA-Specific CAR T-Cells Combined With a Gamma Secretase Inhibitor (JSMD194) to Treat Relapsed or Persistent Multiple Myeloma
- Biomarker Research Study for PF-03084014 in cHEmoresistant Triple-negative Breast cAncer
- Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies
- Evaluate the Effects of Famotidine or Rabeprazole on the PK of Nirogacestat
- Evaluation of the Response and Non-response of Nirogacestat in Desmoid Tumors- Clinical Study
- Gamma Secretase Inhibitor PF-03084014 in Treating Patients With AIDS-Associated Kaposi Sarcoma
- Gamma-Secretase Inhibitor RO4929097 and Cediranib Maleate in Treating Patients With Advanced Solid Tumors
- Gamma-secretase/Notch Signalling Pathway Inhibitor RO4929097 in Combination With Cisplatin, Vinblastine, and Temozolomide in Treating Patients With Recurrent or Metastatic Melanoma
- Gamma-Secretase/Notch Signalling Pathway Inhibitor RO4929097 in Treating Patients With Advanced Solid Tumors

### In development for (8)

- [Alzheimer disease](https://trialight.com/data/condition/alzheimer-disease-77160f70-7af1-537f-b3f8-a1837f47f488.md) Phase 2
- [Breast cancer](https://trialight.com/data/condition/breast-cancer-8ac10da3-93bd-54c5-a93b-265bc6699689.md) Phase 2
- [Breast neoplasm](https://trialight.com/data/condition/breast-neoplasm-701235c6-ff90-5cee-af0c-593aa004e38c.md) Phase 2
- [Desmoid tumor](https://trialight.com/data/condition/desmoid-tumor-ba8c4931-d9d2-5438-88c9-5cc562a19815.md) Phase 3
- Exocrine pancreatic carcinoma Phase 2
- Fibromatosis Phase 2
- Neoplasm Phase 3
- [Plasma cell myeloma](https://trialight.com/data/condition/plasma-cell-myeloma-37186689-2591-5775-9042-2f2bf046a2b5.md) Phase 2

### Targets (6)

- [APH1A](https://trialight.com/data/target/aph1a-cdd07d6c-f2a0-5c2c-ac76-848b59414af3.md) Gamma-secretase inhibitor
- [APH1B](https://trialight.com/data/target/aph1b-bc67830e-db7a-5f07-a9ff-72ce1b1161a6.md) Gamma-secretase inhibitor
- [NCSTN](https://trialight.com/data/target/ncstn-60c86f5f-caa9-5ea4-b4a4-21ba2bdf4eb0.md) Gamma-secretase inhibitor
- [PSEN1](https://trialight.com/data/target/psen1-f9b214da-651f-571e-a4ac-c5927d77d12a.md) Gamma-secretase inhibitor
- [PSEN2](https://trialight.com/data/target/psen2-c4cb79da-7086-5973-8fac-76c58d524c87.md) Gamma-secretase inhibitor
- [PSENEN](https://trialight.com/data/target/psenen-a97c4365-a6bd-511e-bf83-16ef20009315.md) Gamma-secretase inhibitor

---

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