Nirogacestat
Highest phase reached
Approved
Drug type
Small molecule
First approval year
2023
- First approval year
- 2023
- Listing at source
- Listed
- Highest phase reached
- Approved
- Drug type
- Small molecule
- Drug name
- Nirogacestat
- Molecular weight
- 489.66
- Structure (SMILES)
- CCC[C@H](N[C@H]1CCc2cc(F)cc(F)c2C1)C(=O)Nc1cn(C(C)(C)CNCC(C)(C)C)cn1
- 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
- ATC classification codes
- L01XX81
Sources for recorded values
- CAS registry numbers
- 1290543-63-3
- ChEMBL ID
- CHEMBL1770916
- InChIKey
- VFCRKLWBYMDAED-REWPJTCUSA-N
- PubChem compound IDs
- 46224413
- RxNorm IDs (RxCUI)
- 2670631
- FDA ingredient codes (UNII)
- QZ62892OFJ
- Applicant
- SPRINGWORKS
- Approval date
- 27 Nov 2023
- Application number
- NDA217677
- 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
- 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 name
- nirogacestat
- Type
- Synthetic organic
- Drug name stem
- -stat
- Approval recorded by Guide to Pharmacology
- Yes
Administration routes
- Oral
- 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
- 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
- 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
- NCT00878189
- NCT02462707
- NCT05879146
- NCT01876251
- NCT02338531
- NCT01981551
- NCT03785964
- NCT04195399
- NCT05949099
- NCT02109445
- NCT02299635
- NCT04093596
- NCT04126200
- NCT04171843
- NCT04722146
- NCT05090566
- NCT05137054
- NCT05259839
- NCT05556798
- Dose
- 150 mg
- Route
- Oral
- How often
- Twice daily
- Swallow tablets whole, do not break, crush, or chew
- Take with or without food
- 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
- Recorded assays
- 366
- Active assays
- 71
Active assay types
- Confirmatory
- Gamma Secretase Inhibitors and Modulators
- Antineoplastic Agents
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
- 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 Phase 2
- Breast cancer Phase 2
- Breast neoplasm Phase 2
- Desmoid tumor Phase 3
- Exocrine pancreatic carcinoma Phase 2
- Fibromatosis Phase 2
- Neoplasm Phase 3
- Plasma cell myeloma Phase 2