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AboutAbout ATTR-CMUrgencySuspectDetectDiagnostic flowchartAbout ATTR-PNUrgencyAwarenessPartnershipStudy DesignStudy DesignAbout ATTR-PNUrgencyAwarenessPartnershipEfficacy & SafetyPivotal efficacyLong-term survivalSubgroup analysisKey secondary endpointsEarly efficacy measuresSafety profileATTR-PNPivotal efficacyPivotal efficacy ALTLong-term efficacyMutations efficacyLong-term survivalSafety profileDosingATTR-CM dosingATTR-PNPivotal efficacyPivotal efficacy ALTLong-term efficacyMutations efficacyLong-term survivalSafety profileMOD/MOARole of TTRATTR-CM MODATTR-CM MOAATTR-PNATTR-PN MODATTR-PN MOASupport & ResourcesMaterialsVideosATTR-PNATTR-PN MODATTR-PN MOA

Vyndaqel 61mg is indicated for the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy

▼ This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Refer to section 4.8 of the SmPC for how to report adverse reactions.

TTR protein plays an important role in the body—it is vital to some biologic functions including the transport of thyroxine and retinol1-5
  • Thyroid hormones play a vital role in regulating the body's metabolism, heart and digestive functions, muscle control, brain development, and maintenance of bones
  • Retinol (also known as Vitamin A) is important for vision, reproduction, growth, and development7  
  • In large cohort studies, low plasma TTR concentrations have been associated with a higher risk of heart failure8*  
  • The long-term impact of TTR reduction in humans is unknown

TTR is a tetrameric protein mainly produced in the liver1

An ex-US study of 16,967 participants with consecutively ascertained plasma transthyretin concentration in 2 similar prospective studies of the general population. The Cox regression method was used to examine the association between genetically lower transthyretin concentration and incident heart failure.8 TTR=transthyretin.
Explore MoreVYNDAQEL specifically targets TTR destabilization
View VYNDAQEL Mechanism of Action
NEXT: ATTR-CM mechanism of disease
References:Vieira M, Saraiva MJ. Transthyretin: a multifaceted protein. Biomol Concepts. 2014;5(1):45-54. doi:10.1515/bmc-2013-0038Ackermann EJ, Guo S, Benson MD, et al. Suppressing transthyretin production in mice, monkeys and humans using 2nd-generation antisense oligonucleotides. Amyloid. 2016;23(3):148-157. doi:10.1080/13506129.2016.1191458Adams D, Cauquil C, Labeyrie C, Beaudonnet G, Algalarrondo V, Theaudin M. TTR kinetic stabilizers and TTR gene silencing: a new era in therapy for familial amyloidotic polyneuropathies. Expert Opin Pharmacother. 2016;17(6):791-802. doi:10.1517/14656566.2016.1145664Santos SD, Lambertsen KL, Clausen BH, Cerqueira JJ, Sousa N, Palha JA. CSF transthyretin neuroprotection in a mouse model of brain ischemia. J Neurochem. 2010;155(6):1434-1444. doi:10.1111/j.1471-4159.2010.07047.xSousa JC, Marques F, Dias-Ferreira E, et al. Transthyretin influences spatial reference memory. Neurobiol Learn Mem. 2007;88(3):381-385. doi: 10.1016/j.nlm.2007.07.006 Shahid MA, Ashraf MA, Sharma S. Physiology, Thyroid Hormone. [Updated 2020 May 18]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2021. Available from: Accessed January 14, 2022.Bar-El Dadon S, Reiten R. Vitamin A and the epigenome. Crit Rev Food Sci Nutr. 2017;57(11):2404-2411. doi:10.1080/10408398.2015.1060940Greve AM, Christoffersen M, Frikke-Schmidt R, Nordestgaard BG, Tybjrerg-Hansen A. Association of low plasma transthyretin concentration with risk of heart failure in the general population. JAMA Cardiol. 2021;6(3):258-266. doi:10.1001 /jamacardio.2020.5969Liz MA, Coelho T, Bellotti V, Fernandez-Arias Ml, Mallaina P, Obici L. A narrative review of the role of transthyretin in health and disease. Neurol Ther. 2020;9(2):395-402. doi:10.1007 /s40120-020-00217-0
VYNDAQEL Prescribing InformationLoading

Legal Category: S1A.Further information available upon request

PP-VYN-IRL-0176. December 2022

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