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Chemistry

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Also known as: Lnp023, 1644670-37-0, Lnp-023, Iptacopan [inn], Iptacopan [usan], Nvp-lnp023-nx
Molecular Formula
C25H30N2O4
Molecular Weight
422.5  g/mol
InChI Key
RENRQMCACQEWFC-UGKGYDQZSA-N
FDA UNII
8E05T07Z6W

Iptacopan Hydrochloride
Iptacopan is an orally available, small-molecule inhibitor of complement factor B (FB) with potential immunomodulatory activity. Upon administration, iptacopan binds to FB and prevents the formation of the alternative pathway (AP) C3-convertase (C3bBb). This limits the cleavage of C3 to the active fragment C3b and may prevent C3b-mediated extravascular hemolysis in certain complement-driven disorders such as paroxysmal nocturnal hemoglobinuria (PNH).
1 2D Structure

Iptacopan Hydrochloride

2 Identification
2.1 Computed Descriptors
2.1.1 IUPAC Name
4-[(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl]benzoic acid
2.1.2 InChI
InChI=1S/C25H30N2O4/c1-4-31-19-10-12-27(22(14-19)17-5-7-18(8-6-17)25(28)29)15-21-20-9-11-26-24(20)16(2)13-23(21)30-3/h5-9,11,13,19,22,26H,4,10,12,14-15H2,1-3H3,(H,28,29)/t19-,22-/m0/s1
2.1.3 InChI Key
RENRQMCACQEWFC-UGKGYDQZSA-N
2.1.4 Canonical SMILES
CCOC1CCN(C(C1)C2=CC=C(C=C2)C(=O)O)CC3=C(C=C(C4=C3C=CN4)C)OC
2.1.5 Isomeric SMILES
CCO[C@H]1CCN([C@@H](C1)C2=CC=C(C=C2)C(=O)O)CC3=C(C=C(C4=C3C=CN4)C)OC
2.2 Other Identifiers
2.2.1 UNII
8E05T07Z6W
2.3 Synonyms
2.3.1 Depositor-Supplied Synonyms

1. Lnp023

2. 1644670-37-0

3. Lnp-023

4. Iptacopan [inn]

5. Iptacopan [usan]

6. Nvp-lnp023-nx

7. Nvp-lnp023

8. 8e05t07z6w

9. 4-[(2s,4s)-4-ethoxy-1-[(5-methoxy-7-methyl-1h-indol-4-yl)methyl]piperidin-2-yl]benzoic Acid

10. 4-[(2~{s},4~{s})-4-ethoxy-1-[(5-methoxy-7-methyl-1~{h}-indol-4-yl)methyl]piperidin-2-yl]benzoic Acid

11. Benzoic Acid, 4-((2s,4s)-4-ethoxy-1-((5-methoxy-7-methyl-1h-indol-4-yl)methyl)-2-piperidinyl)-

12. Benzoic Acid, 4-[(2s,4s)-4-ethoxy-1-[(5-methoxy-7-methyl-1h-indol-4-yl)methyl]-2-piperidinyl]-

13. Iptacopan [who-dd]

14. Unii-8e05t07z6w

15. Chembl4594448

16. Schembl16400416

17. Gtpl10710

18. Us9682968, Example-26a

19. Bdbm160475

20. Ex-a5728

21. Who 11259

22. Zinc223246892

23. At30389

24. Compound 41 [pmid: 32073845]

25. Hy-127105

26. Cs-0093107

27. A935227

28. 4-((2s,4s)-(4-ethoxy-1-((5-methoxy-7-methyl-1h-indol-4-yl)methyl)piperidin-2-yl))benzoic Acid

29. 4-((2s,4s)-4-ethoxy-1-((5-methoxy-7-methyl-1h-indol-4-yl)methyl)piperidin-2-yl)benzoicacid

30. Jgq

2.4 Create Date
2015-02-16
3 Chemical and Physical Properties
Molecular Weight 422.5 g/mol
Molecular Formula C25H30N2O4
XLogP31.8
Hydrogen Bond Donor Count2
Hydrogen Bond Acceptor Count5
Rotatable Bond Count7
Exact Mass422.22055744 g/mol
Monoisotopic Mass422.22055744 g/mol
Topological Polar Surface Area74.8 Ų
Heavy Atom Count31
Formal Charge0
Complexity594
Isotope Atom Count0
Defined Atom Stereocenter Count2
Undefined Atom Stereocenter Count0
Defined Bond Stereocenter Count0
Undefined Bond Stereocenter Count0
Covalently Bonded Unit Count1

NDC API

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IPTACOPAN HYDROCHLORIDE

NDC Package Code : 44139-0100

Start Marketing Date : 2023-12-05

End Marketing Date : 2025-12-31

Dosage Form (Strength) : POWDER (1kg/kg)

Marketing Category : BULK INGREDIENT

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Drugs in Development

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Details:

Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway. It is being evaluated for the treatment of patients with IgA nephropathy (IgAN).


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Nephrology Brand Name: Fabhalta

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 15, 2024

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01

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway. It is being evaluated for the treatment of patients with IgA nephropathy (IgAN).

Brand Name : Fabhalta

Molecule Type : Small molecule

Upfront Cash : Not Applicable

April 15, 2024

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Details:

Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway. It is being evaluated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: Fabhalta

Study Phase: ApprovedProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable March 22, 2024

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02

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway. It is being evaluated for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who have hemolytic anemia.

Brand Name : Fabhalta

Molecule Type : Small molecule

Upfront Cash : Not Applicable

March 22, 2024

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Details:

LNP023 (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway being developed in Phase 3 for patients with C3 glomerulopathy, which met its primary endpoint by providing clinically meaningful and statistically significant proteinuria reduction.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Nephrology Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 11, 2023

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03

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (iptacopan), an oral, Factor B inhibitor of the alternative complement pathway being developed in Phase 3 for patients with C3 glomerulopathy, which met its primary endpoint by providing clinically meaningful and statistically significant proteinu...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

December 11, 2023

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Details:

FDA approved Fabhalta® (iptacopan), a Factor B inhibitor of the alternative complement pathway 15-17, as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: Fabhalta

Study Phase: ApprovedProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 05, 2023

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04

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : FDA approved Fabhalta® (iptacopan), a Factor B inhibitor of the alternative complement pathway 15-17, as the first oral monotherapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.

Brand Name : Fabhalta

Molecule Type : Small molecule

Upfront Cash : Not Applicable

December 05, 2023

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Details:

LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Nephrology Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 02, 2023

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05

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysi...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

October 02, 2023

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Details:

LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable April 26, 2023

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06

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysi...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

April 26, 2023

blank

Details:

LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 13, 2022

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07

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysi...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

December 13, 2022

blank

Details:

LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable December 08, 2022

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08

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of the alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysi...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

December 08, 2022

blank

Details:

LNP023 (Iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in PNH.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Rare Diseases and Disorders Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable October 24, 2022

blank

09

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (Iptacopan) is an investigational first-in-class, orally administered targeted factor B inhibitor of alternative complement pathway. It acts upstream of the C5 terminal pathway, preventing not only intravascular but also extravascular hemolysis in...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

October 24, 2022

blank

Details:

LNP023 (Iptacopan ), an oral, selective factor B inhibitor in patients with C3 glomerulopathy met primary endpoints in both patient cohorts. Iptacopan showed a favorable safety and tolerability profile in the Phase II final analysis, with no serious adverse events.


Lead Product(s): Iptacopan Hydrochloride

Therapeutic Area: Nephrology Brand Name: LNP023

Study Phase: Phase IIIProduct Type: Small molecule

Sponsor: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable November 04, 2021

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10

ASC 2024
Not Confirmed
ASC 2024
Not Confirmed

Details : LNP023 (Iptacopan ), an oral, selective factor B inhibitor in patients with C3 glomerulopathy met primary endpoints in both patient cohorts. Iptacopan showed a favorable safety and tolerability profile in the Phase II final analysis, with no serious adve...

Brand Name : LNP023

Molecule Type : Small molecule

Upfront Cash : Not Applicable

November 04, 2021

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FDA Orange Book

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Switzerland
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IPTACOPAN HYDROCHLORIDE

Brand Name : FABHALTA

Dosage Form : CAPSULE;ORAL

Dosage Strength : EQ 200MG BASE

Approval Date : 2023-12-05

Application Number : 218276

RX/OTC/DISCN : RX

RLD : Yes

TE Code :

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Patents & EXCLUSIVITIES

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US Patents

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IPTACOPAN HYDROCHLORIDE

US Patent Number : 11951101

Drug Substance Claim :

Drug Product Claim :

Application Number : 218276

Patent Use Code : U-3895

Delist Requested :

Patent Use Description :

Patent Expiration Date : 2041-07-15

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IPTACOPAN HYDROCHLORIDE

US Patent Number : 11723901

Drug Substance Claim :

Drug Product Claim :

Application Number : 218276

Patent Use Code : U-3980

Delist Requested :

Patent Use Description :

Patent Expiration Date : 2038-08-30

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Switzerland
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IPTACOPAN HYDROCHLORIDE

US Patent Number : 10093663

Drug Substance Claim :

Drug Product Claim :

Application Number : 218276

Patent Use Code : U-3980

Delist Requested :

Patent Use Description :

Patent Expiration Date : 2034-07-14

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IPTACOPAN HYDROCHLORIDE

US Patent Number : 9682968

Drug Substance Claim : Y

Drug Product Claim : Y

Application Number : 218276

Patent Use Code :

Delist Requested :

Patent Use Description :

Patent Expiration Date : 2034-07-14

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IPTACOPAN HYDROCHLORIDE

US Patent Number : 11603363

Drug Substance Claim : Y

Drug Product Claim : Y

Application Number : 218276

Patent Use Code :

Delist Requested :

Patent Use Description :

Patent Expiration Date : 2041-05-25

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US Exclusivities

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IPTACOPAN HYDROCHLORIDE

Exclusivity Code : NCE

Exclusivity Expiration Date : 2028-12-05

Application Number : 218276

Product Number : 1

Exclusivity Details :

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IPTACOPAN HYDROCHLORIDE

Exclusivity Code : ODE-456

Exclusivity Expiration Date : 2030-12-05

Application Number : 218276

Product Number : 1

Exclusivity Details :

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IPTACOPAN HYDROCHLORIDE

Exclusivity Code : I-949

Exclusivity Expiration Date : 2027-08-07

Application Number : 218276

Product Number : 1

Exclusivity Details :

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