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Current standard of care and emerging treatment strategies
In February 2020, the EMA granted marketing authorisation for tafamidis, a transthyretin stabiliser, available as a once-daily 61 mg oral capsule, for the treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).1 Other available treatment options for ATTR-CM are aimed at symptom management, supportive care including pain management, nutritional and mobility support and mitigation of the effects of the disease on other organs.2
Additional treatment options and currently under investigation. Emerging treatment strategies are aimed at reducing the formation of the amyloid fibrils by targeting different stages of their development.3
These are investigational approaches, and no conclusions can be made about safety and efficacy.
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- Vyndaqel Summary of Product Characterists. Available at: https://www.medicines.org.uk/emc/product/11141/smpc. Last accessed May 2020.
- Hawkins PN, Ando Y, Dispenzen A et al. Evolving landscape in the management of transthyretin amyloidosis. Ann Med. 2015; 47: 625–638.
- Ruberg FL, Grogan M, Hanna M et al. Transthyretin Amyloid Cardiomyopathy. JACC State-of-the-Art Review. JACC. 2019; 73(22): 2872–2891.
- Maurer MS, Elliott P, Comenzo R et al. Addressing Common Questions Encountered in the Diagnosis and Management of Cardiac Amyloidosis. Circulation. 2017; 135(14): 1357–1377.
- Nativi-Nicolau J, Maurer MS. Amyloidosis cardiomyopathy: update in the diagnosis and treatment of the most common types. Curr Opin Cardiol. 2018; 33(5): 571–579.
- Judge DP, Heitner SB, Falk RH et al. Transthyretin Stabilization by AG10 in Symptomatic Transthyretin Amyloid Cardiomyopathy. J Am Coll Cardiol. 2019; 74(3): 285–295.
PP-VYN-GBR-0271. June 2020
Click here for Vyndaqel ▼ (tafamidis) prescribing information