Iptacopan: Difference between revisions

Iptacopan
Clinical data
Trade names	Fabhalta
Other names	LNP023
AHFS/Drugs.com	Fabhalta
License data	US DailyMed: Iptacopan;
Routes of; administration	By mouth
Drug class	Complement factor B inhibitor
ATC code	None;
Legal status
Legal status	US: ℞-only;
Identifiers
CAS Number	1644670-37-0;
PubChem CID	90467622;
DrugBank	DB16200;
ChemSpider	75533872;
UNII	8E05T07Z6W;
KEGG	D12251; D12252;
ChEMBL	ChEMBL4594448;
PDB ligand	JGQ (PDBe, RCSB PDB);
Chemical and physical data
Formula	C25H30N2O4
Molar mass	422.525 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES O=C(O)C1=CC=C([C@H]2N(CC3=C(OC)C=C(C)C4=C3C=CN4)CC[C@H](OCC)C2)C=C1;
	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; Key:RENRQMCACQEWFC-UGKGYDQZSA-N;

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Revision as of 00:32, 12 December 2023

Iptacopan , sold under the brand name Fabhalta, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria.^[1] It is a complement factor B inhibitor that was developed by Novartis.^[1] It is taken by mouth.^[1]

Iptacopan was approved by the US Food and Drug Administration (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023.^[2]^[3]

Medical uses

Iptacopan is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria.^[1]^[4]

Side effects

The FDA label for iptacopan contains a black box warning for the risk of serious and life-threatening infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Neisseria meningitidis, and Haemophilus influenzae type B.^[1]

Research

In a clinical study with twelve participants, iptacopan as a single drug led to the normalization of hemolytic markers in most patients, and no serious adverse events occurred during the 12-week study.^[5]^[6]

Iptacopan is also investigated as a drug in other complement-mediated diseases, like age-related macular degeneration and some types of glomerulopathies.^[7]

References

^ ^a ^b ^c ^d ^e ^f "Fabhalta- iptacopan capsule". DailyMed. 5 December 2023. Retrieved 10 December 2023.
^ "Novartis receives FDA approval for Fabhalta (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH". Novartis (Press release). Retrieved 6 December 2023.
^ "Novel Drug Approvals for 2023". U.S. Food and Drug Administration (FDA). 6 December 2023. Retrieved 10 December 2023.
^ https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/218276Orig1s000ltr.pdf This article incorporates text from this source, which is in the public domain.
^ Jang JH, Wong L, Ko BS, Yoon SS, Li K, Baltcheva I, et al. (August 2022). "Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study". Blood Advances. 6 (15): 4450–4460. doi:10.1182/bloodadvances.2022006960. PMC 9636331. PMID 35561315.{{cite journal}}: CS1 maint: overridden setting (link)
^ "Novartis Phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients". Novartis (Press release). Retrieved 6 September 2023.
^ Schubart A, Anderson K, Mainolfi N, Sellner H, Ehara T, Adams CM, et al. (April 2019). "Small-molecule factor B inhibitor for the treatment of complement-mediated diseases". Proceedings of the National Academy of Sciences of the United States of America. 116 (16): 7926–7931. Bibcode:2019PNAS..116.7926S. doi:10.1073/pnas.1820892116. PMC 6475383. PMID 30926668.{{cite journal}}: CS1 maint: overridden setting (link)

External links

Clinical trial number NCT04558918 for "Study of Efficacy and Safety of Twice Daily Oral LNP023 in Adult PNH Patients With Residual Anemia Despite Anti-C5 Antibody Treatment (APPLY-PNH)" at ClinicalTrials.gov
Clinical trial number NCT04820530 for "Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy (APPOINT-PNH)" at ClinicalTrials.gov

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[Fabhalta_FDA_label-1] ^ ^a ^b ^c ^d ^e ^f "Fabhalta- iptacopan capsule". DailyMed. 5 December 2023. Retrieved 10 December 2023.

[2] "Novartis receives FDA approval for Fabhalta (iptacopan), offering superior hemoglobin improvement in the absence of transfusions as the first oral monotherapy for adults with PNH". Novartis (Press release). Retrieved 6 December 2023.

[3] "Novel Drug Approvals for 2023". U.S. Food and Drug Administration (FDA). 6 December 2023. Retrieved 10 December 2023.

[4] ttps://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/218276Orig1s000ltr.pdf This article incorporates text from this source, which is in the public domain.

[blood_adv-5] Jang JH, Wong L, Ko BS, Yoon SS, Li K, Baltcheva I, et al. (August 2022). "Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study". Blood Advances. 6 (15): 4450–4460. doi:10.1182/bloodadvances.2022006960. PMC 9636331. PMID 35561315.{{cite journal}}: CS1 maint: overridden setting (link)

[6] "Novartis Phase III APPOINT-PNH trial shows investigational oral monotherapy iptacopan improves hemoglobin to near-normal levels, leading to transfusion independence in all treatment-naïve PNH patients". Novartis (Press release). Retrieved 6 September 2023.

[7] Schubart A, Anderson K, Mainolfi N, Sellner H, Ehara T, Adams CM, et al. (April 2019). "Small-molecule factor B inhibitor for the treatment of complement-mediated diseases". Proceedings of the National Academy of Sciences of the United States of America. 116 (16): 7926–7931. Bibcode:2019PNAS..116.7926S. doi:10.1073/pnas.1820892116. PMC 6475383. PMID 30926668.{{cite journal}}: CS1 maint: overridden setting (link)

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 | licence_CA        = <!-- Health Canada may use generic or brand name (generic name preferred) -->