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Leriglitazone
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| Other names | Hydroxypioglitazone |
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| Formula | C19H20N2O4S |
| Molar mass | 372.44 g·mol−1 |
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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
[edit]Legal status
[edit]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
[edit]- ^ 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.
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