Enasidenib
{{Short description|Chemical compound}}
{{Use dmy dates|date=August 2024}}
{{cs1 config |name-list-style=vanc |display-authors=6}}
{{Infobox drug
| image = Enasidenib.svg
| width =
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| caption =
| pronounce =
| tradename = Idhifa
| Drugs.com = {{drugs.com|monograph|enasidenib-mesylate}}
| MedlinePlus = a617040
| DailyMedID = Enasidenib
| pregnancy_AU = D
| pregnancy_AU_comment =
| pregnancy_category=
| routes_of_administration = By mouth
| class = IDH2 inhibitor
| ATC_prefix = L01
| ATC_suffix = XM01
| ATC_supplemental =
| legal_AU = S4
| legal_AU_comment =
| legal_BR =
| legal_BR_comment =
| legal_CA = Rx-only
| legal_CA_comment = {{cite web | title=Summary Basis of Decision (SBD) for Idhifa | website=Health Canada | date=23 October 2014 | url=https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00441&lang=en | access-date=29 May 2022 | archive-date=8 August 2024 | archive-url=https://web.archive.org/web/20240808060517/https://hpr-rps.hres.ca/reg-content/summary-basis-decision-detailTwo.php?linkID=SBD00441&lang=en | url-status=live }}
| legal_DE =
| legal_DE_comment =
| legal_NZ =
| legal_NZ_comment =
| legal_UK =
| legal_UK_comment =
| legal_US = Rx-only
| legal_EU =
| legal_EU_comment =
| legal_UN =
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| bioavailability =
| protein_bound =
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| index2_label = as salt
| CAS_number_Ref =
| CAS_number = 1446502-11-9
| CAS_supplemental =
| PubChem = 89683805
| IUPHAR_ligand =
| DrugBank_Ref =
| DrugBank = DB13874
| ChemSpiderID_Ref =
| ChemSpiderID = 38772329
| UNII_Ref = {{fdacite|correct|FDA}}
| UNII = 3T1SS4E7AG
| KEGG_Ref =
| KEGG = D10901
| KEGG2_Ref =
| KEGG2 = D11044
| ChEBI_Ref =
| ChEBI = 145374
| ChEMBL_Ref =
| ChEMBL = 3989908
| NIAID_ChemDB =
| PDB_ligand = 69Q
| synonyms = AG-221
| C=19 | H=17 | F=6 | N=7 | O=1
| SMILES = CC(C)(CNC1=NC(=NC(=N1)NC2=CC(=NC=C2)C(F)(F)F)C3=NC(=CC=C3)C(F)(F)F)O
| StdInChI = InChI=1S/C19H17F6N7O/c1-17(2,33)9-27-15-30-14(11-4-3-5-12(29-11)18(20,21)22)31-16(32-15)28-10-6-7-26-13(8-10)19(23,24)25/h3-8,33H,9H2,1-2H3,(H2,26,27,28,30,31,32)
| StdInChIKey = DYLUUSLLRIQKOE-UHFFFAOYSA-N
}}
Enasidenib, sold under the brand name Idhifa, is an anti-cancer medication used to treat relapsed or refractory acute myeloid leukemia.{{cite journal | vauthors = Kim ES | s2cid = 7685848 | title = Enasidenib: First Global Approval | journal = Drugs | volume = 77 | issue = 15 | pages = 1705–1711 | date = October 2017 | pmid = 28879540 | doi = 10.1007/s40265-017-0813-2 }} It is an inhibitor of isocitrate dehydrogenase 2 (IDH2).
Common side effects of enasidenib include nausea, vomiting, diarrhea, increased levels of bilirubin (substance found in bile), and decreased appetite. Women who are pregnant or breastfeeding should not take enasidenib because it may cause harm to a developing fetus or a newborn baby.
Enasidenib was approved for medical use in the United States in August 2017.{{cite press release | title=FDA approves new targeted treatment for relapsed or refractory acute myeloid leukemia | website=U.S. Food and Drug Administration | date=1 August 2017 | url=https://www.fda.gov/news-events/press-announcements/fda-approves-new-targeted-treatment-relapsed-or-refractory-acute-myeloid-leukemia | access-date=8 August 2024 | archive-date=2 August 2024 | archive-url=https://web.archive.org/web/20240802083644/https://www.fda.gov/news-events/press-announcements/fda-approves-new-targeted-treatment-relapsed-or-refractory-acute-myeloid-leukemia | url-status=live }} {{PD-notice}} The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.{{cite report | title=New Drug Therapy Approvals 2017 | website=U.S. Food and Drug Administration (FDA) | date=January 2018 | url=https://www.fda.gov/media/110526/download | format=PDF | access-date=16 September 2020 | archive-date=23 October 2020 | archive-url=https://web.archive.org/web/20201023035728/https://www.fda.gov/media/110526/download | url-status=live }}
Medical use
Enasidenib is indicated for the treatment of relapsed or refractory acute myeloid leukemia in adults with specific mutations of the IDH2 gene, determined by an FDA-approved IDH2 companion diagnostic test.
Adverse effects
Common side effects of enasidenib include nausea, vomiting, diarrhea, increased levels of bilirubin (substance found in bile) and decreased appetite. Women who are pregnant or breastfeeding should not take enasidenib because it may cause harm to a developing fetus or a newborn baby. The prescribing information for enasidenib includes a boxed warning that an adverse reaction known as differentiation syndrome can occur and can be fatal if not treated.{{Cite book|title=Goodman & Gilman's the pharmacological basis of therapeutics|date=5 December 2017 | vauthors = Brunton LL, Hilal-Dandan R, Knollmann BC |isbn=9781259584732|edition=13th|location=New York |publisher=McGraw Hill Education |oclc=993810322}}
History
The efficacy of enasidenib was studied in a single-arm trial of 199 participants with relapsed or refractory acute myeloid leukemia who had isocitrate dehydrogenase-2 mutations as detected by the RealTime IDH2 Assay. The trial measured the percentage of participants with no evidence of disease and full recovery of blood counts after treatment (complete remission), as well as participants with no evidence of disease and partial recovery of blood counts after treatment (complete remission with partial hematologic recovery). With a minimum of six months of treatment, 19 percent of participants experienced complete remission for a median 8.2 months, and 4 percent of participants experienced complete remission with partial hematologic recovery for a median 9.6 months. Of the 157 participants who required transfusions of blood or platelets due to acute myeloid leukemia at the start of the study, 34 percent no longer required transfusions after treatment with enasidenib.
The US Food and Drug Administration (FDA) granted the application for enasidenib priority review and orphan drug designations.
Society and culture
= Legal status =
Enasidenib was approved by the FDA in August 2017, for relapsed or refractory acute myeloid leukemia (AML) in people with specific mutations of the IDH2 gene, determined by an FDA-approved IDH2 companion diagnostic test.
References
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External links
- {{cite web | work=NCI Dictionary of Cancer Terms | publisher=National Cancer Institute | url=https://www.cancer.gov/publications/dictionaries/cancer-terms/def/enasidenib-mesylate | title=Enasidenib mesylate }}
- {{cite web | title=Enasidenib mesylate | website=National Cancer Institute | date=8 August 2017 | url=https://www.cancer.gov/about-cancer/treatment/drugs/enasidenibmesylate }}
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