Tafamidis meglumine

• CAT Number: I014697
• CAS Number: 951395-08-7 (meglumine)
• Molecular Formula: C21H24Cl2N2O8
• Molecular Weight: 503.329
• Purity: ≥95%
• Solubility: Soluble in DMSO
• InChI: InChI=1S/C14H7Cl2NO3.C7H17NO5/c15-9-3-8(4-10(16)6-9)13-17-11-2-1-7(14(18)19)5-12(11)20-13;1-8-2-4(10)6(12)7(13)5(11)3-9/h1-6H,(H,18,19);4-13H,2-3H2,1H3/t;4-,5+,6+,7+/m.0/s1
• InChIKey: DQJDBUPLRMRBAB-WZTVWXICSA-N
• SMILES: OC[C@@H](O)[C@@H](O)[C@H](O)[C@@H](O)CNC.O=C(C1=CC=C2N=C(C3=CC(Cl)=CC(Cl)=C3)OC2=C1)O

Tafamidis meglumine(CAT: I014697) signifies a pivotal development in the field of pharmaceutical research and the treatment of certain diseases. Tafamidis, in its meglumine salt form, is a medication approved for specific amyloid diseases. It serves as a transthyretin (TTR) stabilizer, working to inhibit the formation of amyloid fibrils. By targeting TTR, a protein involved in transporting thyroid hormones and retinol-binding protein, Tafamidis meglumine aims to slow the progression of hereditary transthyretin-mediated amyloidosis (hATTR) and delay associated neurological and cardiac complications.

Reference

1: Ikeda T, Masuda T, Ueda M, Yamashita T, Misumi Y, Shinriki S, Ando Y. Unwanted road to anaemia in transthyretin familial amyloid polyneuropathy may continue irrespective of tafamidis treatment. Ann Clin Biochem. 2018 Jan 1:4563218754587. doi: 10.1177/0004563218754587. [Epub ahead of print] PubMed PMID: 29310464. 2: Gundapaneni BK, Sultan MB, Keohane DJ, Schwartz JH. Tafamidis delays neurological progression comparably across Val30Met and non-Val30Met genotypes in transthyretin familial amyloid polyneuropathy. Eur J Neurol. 2018 Mar;25(3):464-468. doi: 10.1111/ene.13510. Epub 2017 Dec 26. PubMed PMID: 29115008; PubMed Central PMCID: PMC5838526. 3: Sultan MB, Gundapaneni B, Schumacher J, Schwartz JH. Treatment With Tafamidis Slows Disease Progression in Early-Stage Transthyretin Cardiomyopathy. Clin Med Insights Cardiol. 2017 Sep 18;11:1179546817730322. doi: 10.1177/1179546817730322. eCollection 2017. PubMed PMID: 28951660; PubMed Central PMCID: PMC5606341. 4: Romero-Imbroda J, Sagrario-Fustero T, Del Canto-Pérez C. Tafamidis for a Transplant Patient with Transthyretin Amyloid Polyneuropathy. J Clin Neurol. 2017 Oct;13(4):444-446. doi: 10.3988/jcn.2017.13.4.444. Epub 2017 Sep 4. PubMed PMID: 28884984; PubMed Central PMCID: PMC5653640. 5: Lee KR, Jeong JW, Hyun HC, Jang E, Ahn S, Choi S, Joo SH, Kim S, Koo TS. Pharmacokinetics of tafamidis, a transthyretin amyloidosis drug, in rats. Xenobiotica. 2017 Nov 16:1-8. doi: 10.1080/00498254.2017.1366575. [Epub ahead of print] PubMed PMID: 28803538. 6: Barroso FA, Judge DP, Ebede B, Li H, Stewart M, Amass L, Sultan MB. Long-term safety and efficacy of tafamidis for the treatment of hereditary transthyretin amyloid polyneuropathy: results up to 6 years. Amyloid. 2017 Sep;24(3):194-204. doi: 10.1080/13506129.2017.1357545. Epub 2017 Jul 31. PubMed PMID: 28758793. 7: Maurer MS, Elliott P, Merlini G, Shah SJ, Cruz MW, Flynn A, Gundapaneni B, Hahn C, Riley S, Schwartz J, Sultan MB, Rapezzi C; ATTR-ACT Study Investigators. Design and Rationale of the Phase 3 ATTR-ACT Clinical Trial (Tafamidis in Transthyretin Cardiomyopathy Clinical Trial). Circ Heart Fail. 2017 Jun;10(6). pii: e003815. doi: 10.1161/CIRCHEARTFAILURE.116.003815. Review. PubMed PMID: 28611125. 8: Keohane D, Schwartz J, Gundapaneni B, Stewart M, Amass L. Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial. Amyloid. 2017 Mar;24(1):30-36. doi: 10.1080/13506129.2017.1301419. Epub 2017 Apr 10. PubMed PMID: 28393570. 9: Miyazaki Y. Treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a case report. Nihon Ronen Igakkai Zasshi. 2017;54(1):75-80. doi: 10.3143/geriatrics.54.75. Japanese. PubMed PMID: 28202889. 10: Fujita T, Inomata T, Kaida T, Iida Y, Ikeda Y, Nabeta T, Ishii S, Maekawa E, Naruke T, Koitabashi T, Kitamura E, Sekijima Y, Ako J. Tafamidis for the Treatment of Hereditary Transthyretin Amyloid Cardiomyopathy: A Case Report. Cardiology. 2017;137(2):74-77. doi: 10.1159/000455089. Epub 2017 Feb 3. PubMed PMID: 28152524. 11: Hyun HC, Jeong JW, Kim HR, Oh JH, Lee JH, Choi S, Kim YS, Koo TS. Development and validation of a liquid chromatography-tandem mass spectrometry method for the assay of tafamidis in rat plasma: Application to a pharmacokinetic study in rats. J Pharm Biomed Anal. 2017 Apr 15;137:90-95. doi: 10.1016/j.jpba.2017.01.020. Epub 2017 Jan 10. PubMed PMID: 28107689. 12: Institute for Quality and Efficiency in Health Care. Tafamidis Meglumine — Benefit Assessment According to §35a Social Code Book V [Internet]. Cologne, Germany: Institute for Quality and Efficiency in Health Care (IQWiG); 2012 Mar 13. Available from http://www.ncbi.nlm.nih.gov/books/NBK385651/ PubMed PMID: 27905733. 13: Planté-Bordeneuve V, Gorram F, Salhi H, Nordine T, Ayache SS, Le Corvoisier P, Azoulay D, Feray C, Damy T, Lefaucheur JP. Long-term treatment of transthyretin familial amyloid polyneuropathy with tafamidis: a clinical and neurophysiological study. J Neurol. 2017 Feb;264(2):268-276. doi: 10.1007/s00415-016-8337-3. Epub 2016 Nov 22. PubMed PMID: 27878441. 14: Waddington Cruz M, Amass L, Keohane D, Schwartz J, Li H, Gundapaneni B. Early intervention with tafamidis provides long-term (5.5-year) delay of neurologic progression in transthyretin hereditary amyloid polyneuropathy. Amyloid. 2016 Sep;23(3):178-183. Epub 2016 Aug 5. PubMed PMID: 27494299; PubMed Central PMCID: PMC5359776. 15: Casal I, Monteiro S, Beirão JM. Tafamidis in hereditary ATTR amyloidosis – our experience on monitoring the ocular manifestations. Amyloid. 2016 Dec;23(4):262-263. Epub 2016 Oct 17. PubMed PMID: 27748624. 16: Cortese A, Vita G, Luigetti M, Russo M, Bisogni G, Sabatelli M, Manganelli F, Santoro L, Cavallaro T, Fabrizi GM, ScheNA A, Grandis M, Gemelli C, Mauro A, Pradotto LG, Gentile L, Stancanelli C, Lozza A, Perlini S, Piscosquito G, Calabrese D, Mazzeo A, Obici L, Pareyson D. Erratum to: Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area. J Neurol. 2016 May;263(5):925-926. doi: 10.1007/s00415-016-8116-1. PubMed PMID: 27098978. 17: Cortese A, Vita G, Luigetti M, Russo M, Bisogni G, Sabatelli M, Manganelli F, Santoro L, Cavallaro T, Fabrizi GM, ScheNA A, Grandis M, Gemelli C, Mauro A, Pradotto LG, Gentile L, Stancanelli C, Lozza A, Perlini S, Piscosquito G, Calabrese D, Mazzeo A, Obici L, Pareyson D. Monitoring effectiveness and safety of Tafamidis in transthyretin amyloidosis in Italy: a longitudinal multicenter study in a non-endemic area. J Neurol. 2016 May;263(5):916-924. doi: 10.1007/s00415-016-8064-9. Epub 2016 Mar 16. Erratum in: J Neurol. 2016 May;263(5):925-926. PubMed PMID: 26984605. 18: Ando Y, Sekijima Y, Obayashi K, Yamashita T, Ueda M, Misumi Y, Morita H, Machii K, Ohta M, Takata A, Ikeda S. Effects of tafamidis treatment on transthyretin (TTR) stabilization, efficacy, and safety in Japanese patients with familial amyloid polyneuropathy (TTR-FAP) with Val30Met and non-Val30Met: A phase III, open-label study. J Neurol Sci. 2016 Mar 15;362:266-71. doi: 10.1016/j.jns.2016.01.046. Epub 2016 Jan 22. PubMed PMID: 26944161. 19: Coelho T, Merlini G, Bulawa CE, Fleming JA, Judge DP, Kelly JW, Maurer MS, Planté-Bordeneuve V, Labaudinière R, Mundayat R, Riley S, Lombardo I, Huertas P. Mechanism of Action and Clinical Application of Tafamidis in Hereditary Transthyretin Amyloidosis. Neurol Ther. 2016 Jun;5(1):1-25. doi: 10.1007/s40120-016-0040-x. Epub 2016 Feb 19. Review. PubMed PMID: 26894299; PubMed Central PMCID: PMC4919130. 20: Waddington Cruz M, Benson MD. A Review of Tafamidis for the Treatment of Transthyretin-Related Amyloidosis. Neurol Ther. 2015 Dec;4(2):61-79. doi: 10.1007/s40120-015-0031-3. Epub 2015 Aug 15. PubMed PMID: 26662359; PubMed Central PMCID: PMC4685869.