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'''Iptacopan''' , sold under the brand name '''Fabhalta''', is a medication used for the treatment of [[paroxysmal nocturnal hemoglobinuria]].<ref name="Fabhalta FDA label" /> It is a [[complement factor B]] inhibitor that was developed by [[Novartis]].<ref name="Fabhalta FDA label" /> It is taken [[by mouth]].<ref name="Fabhalta FDA label" />
'''Iptacopan''' , sold under the brand name '''Fabhalta''', is a medication used for the treatment of [[paroxysmal nocturnal hemoglobinuria]].<ref name="Fabhalta FDA label" /> It is a [[complement factor B]] inhibitor that was developed by [[Novartis]].<ref name="Fabhalta FDA label" /> It is taken [[by mouth]].<ref name="Fabhalta FDA label" />


Iptacopan was approved by the US [[Food and Drug Administration]] (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023.<ref>{{Cite press release |title=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 |url=https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh |access-date=2023-12-06 |website=Novartis }}</ref><ref>{{cite web | title=Novel Drug Approvals for 2023 | website=U.S. [[Food and Drug Administration]] (FDA) | date=6 December 2023 | url=https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 | access-date=10 December 2023}}</ref>
Iptacopan was approved by the US [[Food and Drug Administration]] (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023.<ref>{{Cite press release |title=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 |url=https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-fabhalta-iptacopan-offering-superior-hemoglobin-improvement-absence-transfusions-first-oral-monotherapy-adults-pnh |access-date=6 December 2023 |website=Novartis }}</ref><ref>{{cite web | title=Novel Drug Approvals for 2023 | website=U.S. [[Food and Drug Administration]] (FDA) | date=6 December 2023 | url=https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 | access-date=10 December 2023}}</ref>


== Medical uses ==
== Medical uses ==
Line 110: Line 110:


== Research ==
== 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.<ref name=blood_adv>{{cite journal | vauthors = Jang JH, Wong L, Ko BS, Yoon SS, Li K, Baltcheva I, Nidamarthy PK, Chawla R, Junge G, Yap ES | display-authors = 6 | title = Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study | journal = Blood Advances | volume = 6 | issue = 15 | pages = 4450–4460 | date = August 2022 | pmid = 35561315 | doi = 10.1182/bloodadvances.2022006960 | pmc = 9636331 }}</ref><ref>{{Cite press release |title=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 |url=https://www.novartis.com/news/media-releases/novartis-phase-iii-appoint-pnh-trial-shows-investigational-oral-monotherapy-iptacopan-improves-hemoglobin-near-normal-levels-leading-transfusion-independence-all-treatment-naive-pnh-patients |access-date=2023-09-06 |website=Novartis }}</ref>
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.<ref name=blood_adv>{{cite journal | vauthors = Jang JH, Wong L, Ko BS, Yoon SS, Li K, Baltcheva I, Nidamarthy PK, Chawla R, Junge G, Yap ES | display-authors = 6 | title = Iptacopan monotherapy in patients with paroxysmal nocturnal hemoglobinuria: a 2-cohort open-label proof-of-concept study | journal = Blood Advances | volume = 6 | issue = 15 | pages = 4450–4460 | date = August 2022 | pmid = 35561315 | doi = 10.1182/bloodadvances.2022006960 | pmc = 9636331 }}</ref><ref>{{Cite press release |title=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 |url=https://www.novartis.com/news/media-releases/novartis-phase-iii-appoint-pnh-trial-shows-investigational-oral-monotherapy-iptacopan-improves-hemoglobin-near-normal-levels-leading-transfusion-independence-all-treatment-naive-pnh-patients |access-date=6 September 2023 |website=Novartis }}</ref>


Iptacopan is also investigated as a drug in other complement-mediated diseases, like age-related [[macular degeneration]] and some types of [[glomerulopathy|glomerulopathies]].<ref>{{cite journal | vauthors = Schubart A, Anderson K, Mainolfi N, Sellner H, Ehara T, Adams CM, Mac Sweeney A, Liao SM, Crowley M, Littlewood-Evans A, Sarret S, Wieczorek G, Perrot L, Dubost V, Flandre T, Zhang Y, Smith RJ, Risitano AM, Karki RG, Zhang C, Valeur E, Sirockin F, Gerhartz B, Erbel P, Hughes N, Smith TM, Cumin F, Argikar UA, Haraldsson B, Mogi M, Sedrani R, Wiesmann C, Jaffee B, Maibaum J, Flohr S, Harrison R, Eder J | display-authors = 6 | title = Small-molecule factor B inhibitor for the treatment of complement-mediated diseases | journal = Proceedings of the National Academy of Sciences of the United States of America | volume = 116 | issue = 16 | pages = 7926–7931 | date = April 2019 | pmid = 30926668 | doi = 10.1073/pnas.1820892116 | pmc = 6475383 | bibcode = 2019PNAS..116.7926S | doi-access = free }}</ref>
Iptacopan is also investigated as a drug in other complement-mediated diseases, like age-related [[macular degeneration]] and some types of [[glomerulopathy|glomerulopathies]].<ref>{{cite journal | vauthors = Schubart A, Anderson K, Mainolfi N, Sellner H, Ehara T, Adams CM, Mac Sweeney A, Liao SM, Crowley M, Littlewood-Evans A, Sarret S, Wieczorek G, Perrot L, Dubost V, Flandre T, Zhang Y, Smith RJ, Risitano AM, Karki RG, Zhang C, Valeur E, Sirockin F, Gerhartz B, Erbel P, Hughes N, Smith TM, Cumin F, Argikar UA, Haraldsson B, Mogi M, Sedrani R, Wiesmann C, Jaffee B, Maibaum J, Flohr S, Harrison R, Eder J | display-authors = 6 | title = Small-molecule factor B inhibitor for the treatment of complement-mediated diseases | journal = Proceedings of the National Academy of Sciences of the United States of America | volume = 116 | issue = 16 | pages = 7926–7931 | date = April 2019 | pmid = 30926668 | doi = 10.1073/pnas.1820892116 | pmc = 6475383 | bibcode = 2019PNAS..116.7926S | doi-access = free }}</ref>

Revision as of 00:17, 12 December 2023

Iptacopan
Clinical data
Trade namesFabhalta
Other namesLNP023
AHFS/Drugs.comfabhalta
License data
Routes of
administration		By mouth
Drug classComplement factor B inhibitor
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
PubChem CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
PDB ligand
Chemical and physical data
FormulaC25H30N2O4
Molar mass422.525 g·mol−1
3D model (JSmol)
  • 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=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

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

  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. ^ https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2023/218276Orig1s000ltr.pdf Public Domain This article incorporates text from this source, which is in the public domain.
  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)
  • 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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