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2000
Volume 22, Issue 2
  • ISSN: 1871-5257
  • E-ISSN: 1875-6182

Abstract

Background

Cardiomyopathy is a global health crisis that affects people all over the world. Consequently, scientists felt compelled to look for and develop ever-more-powerful pharmaceuticals. For ATTR-CM, the only drug currently recommended by the European Society of Cardiology is Tafamidis.

Objectives

The primary aim of this review article is to understand the chemistry, pharmacodynamic, pharmacokinetic, and bio-analytical methods available for Tafamidis.

Methods

A systematic review of the existing resources was accomplished up to 2022, comprising existing studies forming the database covering the existing resources from Web of Science, ScienceDirect, and PubMed.

Results

The review was based on a systematic review of all the existing studies used to formulate the database. The study also illustrated the PRISMA design that systematically analyses the prevalent resources.

Conclusion

Minimal analytical techniques are observed for quantifying the Tafamidis and transthyretin kinetic stabiliser. Therapeutic, pharmacological, and analytical considerations for the novel drug Tafamidis are discussed in this review. Particular attention is paid to the many different analytical and bioanalytical methods currently available for estimating Tafamidis, and the need is highlighted to develop a straightforward, validated technique that meets green chemistry standards.

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2025-02-17
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References

  1. VermaB. PatelP. Tafamidis.Treasure Island, FLStatPearls Publishing2022
    [Google Scholar]
  2. BurtonA. CastañoA. BrunoM. RileyS. SchumacherJ. SultanM.B. TaiS.S. JudgeD.P. KellyJ.W. Drug discovery and development in rare diseases: Taking a closer look at the tafamidis.202112251243
    [Google Scholar]
  3. MonteiroC. Martins da SilvaA. FerreiraN. MesgarzadehJ. NovaisM. CoelhoT. KellyJ.W. Cerebrospinal fluid and vitreous body exposure to orally administered tafamidis in hereditary ATTRV30M (p.TTRV50M) amyloidosis patients.Amyloid201825212012810.1080/13506129.2018.147924929993288
    [Google Scholar]
  4. ElliottP. DrachmanB.M. GottliebS.S. HoffmanJ.E. HummelS.L. LenihanD.J. EbedeB. GundapaneniB. LiB. SultanM.B. ShahS.J. Circulation : Heart failure long-term survival with tafamidis in patients with transthyretin amyloid cardiomyopathy.2022411
    [Google Scholar]
  5. CruzM.W. Tafamidis for autonomic neuropathy in hereditary transthyretin (ATTR) amyloidosis: A review.Clin. Auton. Res.201929S1)(1192410.1007/s10286‑019‑00625‑931407119
    [Google Scholar]
  6. GiblinG.T. CuddyS.A.M. GonzaE. SewellA. MurphyA. DorbalaS. FalkR.H. Effect of tafamidis on global longitudinal strain and myocardial work in transthyretin cardiac amyloidosis.European Heart Journal - Cardiovascular Imaging20222381029103910.1093/ehjci/jeac049
    [Google Scholar]
  7. MarquesN. AzevedoO. AlmeidaA.R. BentoD. CruzI. CorreiaE. LourençoC. LopesL.R. Specific therapy for transthyretin cardiac amyloidosis: A systematic literature review and evidence‐based recommendations.J. Am. Heart Assoc.2020919e01661410.1161/JAHA.120.01661432969287
    [Google Scholar]
  8. KaziD.S. BellowsB.K. BaronS.J. ShenC. CohenD.J. SpertusJ.A. YehR.W. ArnoldS.V. SperryB.W. MaurerM.S. ShahS.J. Cost-Effectiveness of tafamidis therapy for transthyretin amyloid cardiomyopathy.Circulation2020141151214122410.1161/CIRCULATIONAHA.119.04509332078382
    [Google Scholar]
  9. MagrinelliF. FabriziG.M. SantoroL. ManganelliF. ZanetteG. CavallaroT. TamburinS. Pharmacological treatment for familial amyloid polyneuropathy.Cochrane Database Syst. Rev.202044CD01239532311072
    [Google Scholar]
  10. Cristóbal GutiérrezH. Pelayo-NegroA.L. Gómez GómezD. Martín VegaM.Á. Valero DomínguezM. Overview of treatments used in transthyretin-related hereditary amyloidosis: A systematic review.Eur. J. Hosp. Pharm. Sci. Pract.202027419420110.1136/ejhpharm‑2018‑00182332587078
    [Google Scholar]
  11. CoelhoT. MaiaL.F. da SilvaA.M. CruzM.W. Planté-BordeneuveV. SuhrO.B. ConceiçaoI. SchmidtH.H.J. TrigoP. KellyJ.W. LabaudinièreR. ChanJ. PackmanJ. GroganD.R. Long-term effects of tafamidis for the treatment of transthyretin familial amyloid polyneuropathy.J. Neurol.2013260112802281410.1007/s00415‑013‑7051‑723974642
    [Google Scholar]
  12. AmassL. LiH. GundapaneniB.K. SchwartzJ.H. KeohaneD.J. Influence of baseline neurologic severity on disease progression and the associated disease-modifying effects of tafamidis in patients with transthyretin amyloid polyneuropathy.Orphanet J. Rare Dis.201813122510.1186/s13023‑018‑0947‑730558645
    [Google Scholar]
  13. CoelhoT. MaiaL.F. Martins da SilvaA. Waddington CruzM. Planté-BordeneuveV. LozeronP. SuhrO.B. CampistolJ.M. ConceiçãoI.M. SchmidtH.H.J. TrigoP. KellyJ.W. LabaudinièreR. ChanJ. PackmanJ. WilsonA. GroganD.R. Tafamidis for transthyretin familial amyloid polyneuropathy: A randomized, controlled trial.Neurology201279878579210.1212/WNL.0b013e3182661eb122843282
    [Google Scholar]
  14. GriffinJ.M. RosenblumH. MaurerM.S. Pathophysiology and therapeutic approaches to cardiac amyloidosis.Circ. Res.2021128101554157510.1161/CIRCRESAHA.121.31818733983835
    [Google Scholar]
  15. TschöpeC. ElsanhouryA. Treatment of transthyretin amyloid cardiomyopathy: The current options, the future, and the challenges.J. Clin. Med.2022118214810.3390/jcm1108214835456241
    [Google Scholar]
  16. GertzM.A. DispenzieriA. Systemic amyloidosis recognition, prognosis, and therapy.JAMA20203241798910.1001/jama.2020.549332633805
    [Google Scholar]
  17. DrugN. New drug: Tafamidis and tafamidis meglumine for amyloid cardiomyopathy.Aust. Prescr.202144413910.18773/austprescr.2021.03234421180
    [Google Scholar]
  18. NencettiS. RosselloA. OrlandiniE. Tafamidis (Vyndaqel): A light for FAP patients.ChemMedChem2013810n/a10.1002/cmdc.20130024524000164
    [Google Scholar]
  19. de LartigueJ. Tafamidis for transthyretin amyloidosis.Drugs Today201248533133710.1358/dot.2012.48.5.180848622645721
    [Google Scholar]
  20. European medicines agency, C. for M.P. for H.U. (CHMP).Assessment Report Vyndaqel.2011201144
    [Google Scholar]
  21. Waddington CruzM. BensonM.D. A review of tafamidis for the treatment of transthyretin-related amyloidosis.Neurol. Ther.201542617910.1007/s40120‑015‑0031‑326662359
    [Google Scholar]
  22. Pfizer. Pfizer’s Vyndaqel® (Tafamidis) First Therapy Approved in the European Union for the Rare and Fatal Neurodegenerative Disease Transthyretin Familial Amyloid Polyneuropathy (TTR-FAP).2011Available From: https://www.pfizer.com/news/press-release/press-release-detail/pfizer_s_vyndaqel_tafamidis_first_therapy_approved_in_the_european_union_for_the_rare_and_fatal_neurodegenerative_disease_transthyretin_familial_amyloid_polyneuropathy_ttr_fap
  23. ScottL.J. Tafamidis: A review of its use in familial amyloid polyneuropathy.Drugs201474121371137810.1007/s40265‑014‑0260‑225022953
    [Google Scholar]
  24. MonteiroC. MesgazardehJ.S. AnselmoJ. FernandesJ. NovaisM. RodriguesC. BrightyG.J. PowersD.L. PowersE.T. CoelhoT. KellyJ.W. Predictive model of response to tafamidis in hereditary ATTR polyneuropathy.JCI Insight2019412e12652610.1172/jci.insight.12652631217346
    [Google Scholar]
  25. SerranoD. AtzingerC.B. BottemanM.F. Understanding the disease course and therapeutic benefit of tafamidis across real-world studies of hereditary transthyretin amyloidosis with polyneuropathy: A proof of concept for integrative data analytic approaches.Neurol. Ther.20187114115410.1007/s40120‑018‑0096‑x29611130
    [Google Scholar]
  26. YeeA.W. AldeghiM. BlakeleyM.P. OstermannA. MasP.J. MoulinM. de SanctisD. BowlerM.W. Mueller-DieckmannC. MitchellE.P. HaertleinM. de GrootB.L. Boeri ErbaE. ForsythV.T. A molecular mechanism for transthyretin amyloidogenesis.Nat. Commun.201910192510.1038/s41467‑019‑08609‑z30804345
    [Google Scholar]
  27. DamyT. Garcia-PaviaP. HannaM. JudgeD.P. MerliniG. GundapaneniB. PattersonT.A. RileyS. SchwartzJ.H. SultanM.B. WittelesR. Efficacy and safety of tafamidis doses in the TAFAMIDIS IN TRANSTHYRETIN CARDIOMYOPATHY CLINICAL TRIAL (ATTR‐ACT) and long‐term extension study.Eur. J. Heart Fail.202123227728510.1002/ejhf.202733070419
    [Google Scholar]
  28. QuockT.P. YanT. ChangE. GuthrieS. BroderM.S. Epidemiology of AL amyloidosis: A real-world study using US claims data.Blood Adv.20182101046105310.1182/bloodadvances.201801640229748430
    [Google Scholar]
  29. RubergF.L. GroganM. HannaM. KellyJ.W. MaurerM.S. Transthyretin amyloid cardiomyopathy.J. Am. Coll. Cardiol.201973222872289110.1016/j.jacc.2019.04.00331171094
    [Google Scholar]
  30. KeohaneD. SchwartzJ. GundapaneniB. StewartM. AmassL. Tafamidis delays disease progression in patients with early stage transthyretin familial amyloid polyneuropathy: additional supportive analyses from the pivotal trial.Amyloid2017241303610.1080/13506129.2017.130141928393570
    [Google Scholar]
  31. CoelhoT. MerliniG. BulawaC.E. FlemingJ.A. JudgeD.P. KellyJ.W. MaurerM.S. Planté-BordeneuveV. LabaudinièreR. MundayatR. RileyS. LombardoI. HuertasP. Mechanism of action and clinical application of tafamidis in hereditary transthyretin amyloidosis.Neurol. Ther.20165112510.1007/s40120‑016‑0040‑x26894299
    [Google Scholar]
  32. SinghB.M. BoharaN. GautamK. BasnetM. KCS. KCB. RautA. PhudongA. GautamJ. A systematic review of tafamidis in patients with transthyretin amyloid cardiomyopathy.Cureus202113918221
    [Google Scholar]
  33. ZhangY. JiM. Iodine promoted one-pot synthesis of 2-Aryl benzoxazoles from amidoximes via oxidative cyclization and ring contraction.Eur. J. Org. Chem.20192019457506751010.1002/ejoc.201901468
    [Google Scholar]
  34. YuanS. YuB. LiuH.M. New drug approvals for 2019: Synthesis and clinical applications.Eur. J. Med. Chem.202020511266710.1016/j.ejmech.2020.11266732911308
    [Google Scholar]
  35. KarumanchiK. NatarajanS.K. GaddeS. VanchanagiriK. MoturuK.V.R. A new synthesis of tafamidis via zinc-MsOH mediated reductive cyclisation strategy.J. Chem. Sci.202113324810.1007/s12039‑021‑01910‑9
    [Google Scholar]
  36. National center for biotechnology information.2023Available From: https://pubchem.ncbi.nlm.nih.gov/compound/Tafamidis
  37. Le BrasA. Tafamidis: A new treatment for ATTR cardiomyopathy.Nat. Rev. Cardiol.2018151165265210.1038/s41569‑018‑0089‑630214019
    [Google Scholar]
  38. MaurerM.S. MannD.L. The tafamidis drug development program.JACC Basic Transl. Sci.20183687187310.1016/j.jacbts.2018.10.00130623146
    [Google Scholar]
  39. LeeK.R. JeongJ.W. HyunH.C. JangE. AhnS. ChoiS. JooS.H. KimS. KooT.S. Pharmacokinetics of tafamidis, a transthyretin amyloidosis drug, in rats.Xenobiotica201848883183810.1080/00498254.2017.136657528803538
    [Google Scholar]
  40. BriefI. In brief: Tafamidis (Vyndaqel; Vyndamax) for transthyretin amyloid cardiomyopathy.Med. Lett. Drugs Ther.20206215901631999671
    [Google Scholar]
  41. SmerikarovaM. BozhanovS MaslarskaV. A brief review of analytical methods for the estimation of ttr kinetic stabilizers in pharmaceutical formulations and biological matrices.Int. J. Appl. Pharm.20221421
    [Google Scholar]
  42. SmerikarovaM. BozhanovS. MaslarskaV. TournevI. Determination of tafamidis plasma concentrations in amyloidosis patients with glu89gln mutation by HPLC-UV detection.J. Chromatogr. Sci.202260984084710.1093/chromsci/bmab13234897414
    [Google Scholar]
  43. LockwoodP.A. LeV.H. O’GormanM.T. PattersonT.A. SultanM.B. TankishevaE. WangQ. RileyS. The bioequivalence of tafamidis 61‐mg free acid capsules and tafamidis meglumine 4 × 20‐mg capsules in healthy volunteers.Clin. Pharmacol. Drug Dev.20209784985410.1002/cpdd.78932196976
    [Google Scholar]
  44. HyunH.C. JeongJ.W. KimH.R. OhJ.H. LeeJ.H. ChoiS. KimY.S. KooT.S. 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.2017137909510.1016/j.jpba.2017.01.02028107689
    [Google Scholar]
  45. RapezziC. ElliottP. DamyT. Nativi-NicolauJ. BerkJ.L. VelazquezE.J. BomanK. GundapaneniB. PattersonT.A. SchwartzJ.H. SultanM.B. MaurerM.S. Efficacy of tafamidis in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy.JACC Heart Fail.20219211512310.1016/j.jchf.2020.09.01133309574
    [Google Scholar]
  46. HuberP. FlynnA. SultanM.B. LiH. RillD. EbedeB. GundapaneniB. SchwartzJ.H. A comprehensive safety profile of tafamidis in patients with transthyretin amyloid polyneuropathy.Amyloid201926420320910.1080/13506129.2019.164371431353964
    [Google Scholar]
  47. ÇakarA. Durmuş-TekçeH. ParmanY. Familial amyloid polyneuropathy.Noro Psikiyatri Arsivi201956215015631223250
    [Google Scholar]
  48. AndoY. CoelhoT. BerkJ.L. CruzM.W. EriczonB.G. IkedaS. LewisW.D. ObiciL. Planté-BordeneuveV. RapezziC. SaidG. SalviF. Guideline of transthyretin-related hereditary amyloidosis for clinicians.Orphanet J. Rare Dis.2013813110.1186/1750‑1172‑8‑3123425518
    [Google Scholar]
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