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Chemical compound
Leriglitazone
Other names Hydroxypioglitazone
ATC code
5-[[4-[2-[5-(1-Hydroxyethyl)pyridin-2-yl]ethoxy]phenyl]methyl]-1,3-thiazolidine-2,4-dione
CAS Number
PubChem
CID
DrugBank
ChemSpider
UNII
KEGG
ChEMBL
CompTox Dashboard (
EPA )
Formula C 19 H 20 N 2 O 4 S
Molar mass 372.44 g·mol−1 3D model (
JSmol )
CC(C1=CN=C(C=C1)CCOC2=CC=C(C=C2)CC3C(=O)NC(=O)S3)O
InChI=1S/C19H20N2O4S/c1-12(22)14-4-5-15(20-11-14)8-9-25-16-6-2-13(3-7-16)10-17-18(23)21-19(24)26-17/h2-7,11-12,17,22H,8-10H2,1H3,(H,21,23,24)
Key:OXVFDZYQLGRLCD-UHFFFAOYSA-N
Leriglitazone is a
PPAR-gamma agonist and metabolite of the
glitazone
pioglitazone . It is developed for
adrenomyeloneuropathy , and other neurodegenerative diseases.
[1]
[2]
[3]
[4]
Society and culture
Legal status
In January 2024, the
European Medicines Agency (EMA) recommended the refusal of the marketing authorization for leriglitazone (Nezglyal) requested by Minoryx Therapeutics S.L.
[5] In May 2024, the EMA confirmed its recommendation to refuse marketing authorization for leriglitazone.
[5] Leriglitazone was intended for the treatment of cerebral adrenoleukodystrophy.
[5]
References
^ Musokhranova, Uliana; Grau, Cristina; Vergara, Cristina; Rodríguez-Pascau, Laura; Xiol, Clara; Castells, Alba A.; Alcántara, Soledad; Rodríguez-Pombo, Pilar; Pizcueta, Pilar; Martinell, Marc; García-Cazorla, Angels; Oyarzábal, Alfonso (26 October 2023).
"Mitochondrial modulation with leriglitazone as a potential treatment for Rett syndrome" . Journal of Translational Medicine . 21 (1): 756.
doi :
10.1186/s12967-023-04622-5 .
ISSN
1479-5876 .
PMC
10601217 .
PMID
37884937 .
^ Pizcueta, Pilar; Vergara, Cristina; Emanuele, Marco; Vilalta, Anna; Rodríguez-Pascau, Laura; Martinell, Marc (6 February 2023).
"Development of PPARγ Agonists for the Treatment of Neuroinflammatory and Neurodegenerative Diseases: Leriglitazone as a Promising Candidate" . International Journal of Molecular Sciences . 24 (4): 3201.
doi :
10.3390/ijms24043201 .
ISSN
1422-0067 .
PMC
9961553 .
PMID
36834611 .
^ Rodríguez-Pascau, Laura; Vilalta, Anna; Cerrada, Marc; Traver, Estefania; Forss-Petter, Sonja; Weinhofer, Isabelle; Bauer, Jan; Kemp, Stephan; Pina, Guillem; Pascual, Sílvia; Meya, Uwe; Musolino, Patricia L.; Berger, Johannes; Martinell, Marc; Pizcueta, Pilar (2 June 2021).
"The brain penetrant PPARγ agonist leriglitazone restores multiple altered pathways in models of X-linked adrenoleukodystrophy" . Science Translational Medicine . 13 (596).
doi :
10.1126/scitranslmed.abc0555 .
PMID
34078742 .
S2CID
235305143 .
^ Pandolfo, Massimo; Reetz, Kathrin; Darling, Alejandra; Rodriguez de Rivera, Francisco Javier; Henry, Pierre-Gilles; Joers, James; Lenglet, Christophe; Adanyeguh, Isaac; Deelchand, Dinesh; Mochel, Fanny; Pousset, Françoise; Pascual, Sílvia; Van den Eede, Delphine; Martin-Ugarte, Itziar; Vilà-Brau, Anna; Mantilla, Adriana; Pascual, María; Martinell, Marc; Meya, Uwe; Durr, Alexandra (December 2022).
"Efficacy and Safety of Leriglitazone in Patients With Friedreich Ataxia: A Phase 2 Double-Blind, Randomized Controlled Trial (FRAMES)" . Neurology Genetics . 8 (6): e200034.
doi :
10.1212/NXG.0000000000200034 .
PMC
9747094 .
PMID
36524101 .
^
a
b
c
"Nezglyal EPAR" .
European Medicines Agency . 25 January 2024. Retrieved 3 February 2024 .