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ButtonLoadingButtonLoading 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

Prescribing Information

ATTR-PNATTR-PN MODATTR-PN MOA

Vyndaqel 61mg is indicated for the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy
(ATTR-CM).1

▼ 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.

Early diagnosis and treatment of ATTR-CM are critical to extend life1ATTR-CM is an underdiagnosed cause of heart failure2,3

Prognosis worsens rapidly with continued amyloid deposition, resulting in advancing organ dysfunction, significant reduction in quality of life, and, ultimately, death2,4

Timely diagnosis and treatment matter

Normal, healthy heart vs thickened walls of an ATTR amyloidosis heart

 Normal healthy heart Illustrative representation. ATTR amyloidosis heart Illustrative representation.Once diagnosed, untreated patients with ATTR-CM have a median survival of ~2 to 3.5 years4
It has been shown that the mean time to diagnosis of ATTR-CM was:

6.1 years for wild-type5              5.7 years for hereditary5

6.1 years for wild-type5
5.7 years for hereditary5

Timely diagnosis and treatment of ATTR-CM were predicted to extend mean life expectancy by 5.46 years (wild-type) and 7.76 years (hereditary) compared with delayed diagnosis6
Download Resources for ATTR-CM 
Suspicion and Detection  It is important to clinically differentiate between ATTR and AL5,7

Although there are multiple proteins that can cause amyloidosis, 98% of currently diagnosed cardiac amyloidosis cases result from fibrils composed of AL or ATTR8

 wtATTR-CM

wtATTR-CM is a type of amyloidosis caused by age-related changes in the stability of TTR proteins.2,3,9,11,12 

  • Idiopathic and not considered to be a hereditary disease
  • Most common in older adults, and the average age at diagnosis is >60 years
  • Accounts for the majority of ATTR-CM cases

Common characteristics that may present in patients with wtATTR-CM 

  • Heart failure3,12,13
  • Cardiac arrhythmias, particularly atrial fibrillation3,4,12,13
  • ​​​​​​​History of orthopedic manifestations, such as bilateral carpal tunnel syndrome, lumbar spinal stenosis, and/or biceps tendon rupture14-16
~3.5 years
Median Survival
Once diagnosed, untreated patients have a median survival of approximately 3.5 years4
~60% of patients
Exhibited Carpal Tunnel Syndrome as an Early
​​​​​​​ Manifestation
~60% of patients with wtATTR-CM experience carpal tunnel syndrome as many as 5 to 7 years prior to the start of cardiac manifestations/heart failure17,18
hATTR-CM

hATTR-CM is a type of amyloidosis caused by a mutation in the TTR gene9

hATTR-CM is inherited in an autosomal dominant pattern, meaning only 1 affected relative is required to pass on the mutation19

About 75% of patients with hATTR-CM exhibited cardiomyopathic features of the disease.20

Carpal tunnel syndrome is also common and can often be the initial symptom in more than 30% of hATTR-CM patients, with cardiomyopathy symptoms following as the disease progresses.21

There is great heterogeneity in penetrance data depending on the phenotype, genotype, and environmental factors.22

Overview of hATTR-CM

Patient considerations 

  • Men and women3
  • Symptom onset may occur as early as 50-60 years of age23*

Prognosis

  • Once diagnosed, untreated patients have a median survival of ~2 to 3 years4

Common characteristics that may present in patients with hATTR-CM:

  • Heart failure3
  • Autonomic dysfunction3,23
  • History of bilateral carpal tunnel syndrome3
  • Peripheral dysfunction3

Recognition of cardiac involvement (via NT-proBNP measurements)

  • Increased serum levels of NT-proBNP can reveal cardiac involvement24
Early recognition of cardiac involvement in hATTR-CM is critical. 
If left untreated, it can result in adverse outcomes, including increased mortality.24 
Diagnosis of hATTR-CM in an index patient should prompt 
genetic counseling and testing of relatives25
A targeted approach may enable diagnosis of disease upon the first detectable sign or symptom25 
Age of onset varies depending on the causative mutation and geographic area.AL=immunoglobulin light chain amyloidosis; ATTR=transthyretin amyloidosis; ATTR-CM=transthyretin amyloid cardiomyopathy; hATTR-CM=hereditary transthyretin amyloid cardiomyopathy; NT-proBNP=N-terminal pro-B-type natriuretic peptide; TTR=transthyretin; wtATTR-CM=wild-type transthyretin amyloid cardiomyopathy.
Explore More Pivotal and 5-year data for ATTR-CM View the Body of Evidence
NEXT: Suspect ATTR-CM
References:Elliott P, Drachman BM, Gottlieb SS, et al. Long-term survival with tafamidis in patients with transthyretin amyloid cardiomyopathy. Circ Heart Fail. 2022;153e008193:1-8.Witteles RM, Bokhari S, Damy T, et al. Screening for transthyretin amyloid cardiomyopathy in everyday practice. JACC Heart Fail. 2019;7(8):709-716. doi:10.1016/j.jchf.2019.04.010Maurer MS, Hanna M, Grogan M, et al. Genotype and phenotype of transthyretin cardiac amyloidosis: THAOS (Transthyretin Amyloid Outcome Survey). J Am Coll Cardiol. 2016;68(2):161-172. doi:10.1016/j.jacc.2016.03.596Maurer MS, Elliott P, Comenzo R, Semigran M, Rapezzi C. Addressing common questions encountered in the diagnosis and management of cardiac amyloidosis. Circulation. 2017;135(14):1357-1377. doi:10.1161/CIRCULATIONAHA.116.024438Rozenbaum MH, Large S, Bhambri R, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiol Ther. 2021;10(1):141-159. doi:10.1007/s40119-021-00219-56Rozenbaum MH, Large S, Bhambri R, et al. Estimating the health benefits of timely diagnosis and treatment of transthyretin amyloid cardiomyopathy. J Comp Eff Res. 2021;10(11):927-938. doi:10.2217/cer-2021-00711Rapezzi C, Merlini G, Quarta CC, et al. Systemic cardiac amyloidoses: disease profiles and clinical courses of the 3 main types. Circulation. 2009;120(13):1203-1212. doi 10.1161/CIRCULATIONAHA.108.843334Garcia-Pavia P, Rapezzi C, Adler Y, et al. Diagnosis and treatment of cardiac amyloidosis. A position statement of the European Society of Cardiology Working Group on Myocardial and Pericardial Diseases. Eur J Heart Fail. 2021;23(4):512-526. doi:10.1002/ejhf.2140 Benson MD, Buxbaum JN, Eisenberg DS, et al. Amyloid nomenclature 2018: recommendations by the lnternabonal Society of Amyloidosis (ISA) nomenclature committee. Amyloid. 2018;25(4):215-219. doi:10.1080/13506129.2018.1549825Donnelly JP, Hanna M. Cardiac amyloidosis: An update on diagnosis and treatment. Cleve Clin J Med. 2017;84(12)(suppl 3):12-26. doi:10.3949/ccjm.84.s3.02Ruberg FL, Grogan M, Hanna M, Kelly JW, Maurer MS. Transthyretin amyloid cardiomyopathy: JACC state-of-the-art review. J Am Coll Cardiol. 2019;73(22):2872-2891. doi:10.1016/j.jacc.2019.04.003Connors LH, Sam F, Skinner M, et al. Heart failure resulting from age-related cardiac amyloid disease associated with wild-type transthyretin: a prospective, observational cohort study. Circulation. 2016;133(3):282-290. doi:10.1161/CIRCULATIONAHA.115.018852Pinney JH, Whelan CJ, Petrie A, et al. Senile systemic amyloidosis: clinical features at presentation and outcome. J Am Heart Assoc. 2013;2(2):e000098. doi:10.1161/JAHA.113.000098Nakagawa M, Sekijima Y, Yazaki M, et al. Carpal tunnel syndrome: a common initial symptom of systemic wild-type ATTR (ATTRwt) amyloidosis. Amyloid. 2016;23(1):58-63. doi:10.3109/13506129.2015.1135792Westermark P, Westermark GT, Suhr OB, Berg S. Transthyretin-derived amyloidosis: probably a common cause of lumbar spinal stenosis. Ups J Med Sci. 2014;119(3):223-228. doi:10.3109/03009734.2014.895786Geller HI, Singh A, Alexander KM, et al. Association between ruptured distal biceps tendon and wild-type transthyretin cardiac amyloidosis. JAMA. 2017;318(10):962-963. doi:10.1001/jama.2017.9236Stern LK, Kittleson MM. Updates in cardiac amyloidosis diagnosis and treatment. Curr Oncol Rep. 2021;23(4):47. doi:10.1007/s11912-021-01028-8aus dem Siepen F, Hein S, Prestel S, et al. Carpal tunnel syndrome and spinal canal stenosis: harbingers of transthyretin amyloid cardiomyopathy? Clin Res Cardiol. 2019;108(12):1324-1330. doi:10.1007/s00392-019-01467-1Sekijima Y. Hereditary transthyretin amyloidosis. In: Adam MP, Ardinger HH, Pagon RA, Wallace SE, eds. GeneReviews [Internet]. Seattle, WA: University of Washington; 1993-2019.Rapezzi C, Quarta CC, Obici L, et al. Disease profile and differential diagnosis of hereditary transthyretin-related amyloidosis with exclusively cardiac phenotype: an Italian perspective. Eur Heart J. 2013;34(7):520-528. doi:10.1093/eurheartj/ehs123Kapoor M, Rossor AM, Laura M, Reilly MM. Clinical presentation, diagnosis, and treatment of TTR amyloidosis. J Neuromuscul Dis. 2019;6(2):189-199. doi:10.3233/JND-180371Lahuerta Pueyo C, Aibar Arregui MA, Gracia Gutierrez A, Bueno Juana E, Menao Guillen S. Estimating the prevalence of allelic variants in the transthyretin gene by analysing large-scale sequencing data. Eur J Hum Genet. 2019;27(5):783-791. doi:10.1038/s41431-019-0337-1Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related amyloidoses and the heart: a clinical overview. Nat Rev Cardiol. 2010;7(7):398-408. doi:10.1038/nrcardio.2010.67Klaassen SHC, Tromp J, Nienhuis HLA, et al. Frequency of and prognostic significance of cardiac involvement at presentation in hereditary transthyretin-derived amyloidosis and the value of N-terminal pro-B-type natriuretic peptide. Am J Cardiol. 2018;121(1):107-112. doi:10.1016/j.amjcard.2017.09.029Conceiçao I, Damy T, Romero M, et al. Early diagnosis of ATTR amyloidosis through targeted follow-up of identified carriers of TTR gene mutations. Amyloid. 2019;26(1):3-9. doi:10.1080/13506129.2018.1556156
VYNDAQEL Prescribing InformationLoading
About ATTR-CM

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PP-VYN-IRL-0287. January 2025

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