Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.



In November 2011, tafamidis (Vyndaqel; Pfizer), a small molecule that inhibits the dissociation of transthyretin tetramers, was granted marketing authorization by the European Commission for the treatment of transthyretin amyloidosis in adult patients with stage 1 symptomatic polyneuropathy to delay peripheral neurological impairment.

This is a preview of subscription content, access via your institution

Relevant articles

Open Access articles citing this article.

Access options

Rent or buy this article

Prices vary by article type



Prices may be subject to local taxes which are calculated during checkout

Figure 1: Tafamidis.


  1. Planté-Bordeneuve, V. & Said, G. Familial amyloid polyneuropathy. Lancet Neurol. 10, 1086–1097 (2011).

    Article  Google Scholar 

  2. Johnson, S. M. et al. The transthyretin amyloidoses: from delineating the molecular mechanism of aggregation linked to pathology to a regulatory-agency-approved drug. J. Mol. Biol. 5 Jan 2012 (doi: 10.1016/j.jmb.2011.12.060).

  3. Miroy, G. J. et al. Inhibiting transthyretin amyloid fibril formation via protein stabilization. Proc. Natl Acad. Sci. USA 93, 15051–15056 (1996).

    Article  CAS  Google Scholar 

  4. Razavi, H. et al. Benzoxazoles as transthyretin amyloid fibril inhibitors: synthesis, evaluation, and mechanism of action. Angew. Chem. Int. Ed. 42, 2758–2761 (2003).

    Article  CAS  Google Scholar 

  5. European Medicines Agency (EMA). European Public Assessment Report. EMA website [online], (2011).

  6. Hellman, U. et al. Heterogeneity of penetrance in familial amyloid polyneuropathy, ATTR Val30Met, in the Swedish population. Amyloid 15, 181–186 (2008).

    Article  CAS  Google Scholar 

  7. Ohya, Y. et al. Manifestations of transthyretin-related familial amyloidotic polyneuropathy:long-term follow-up of Japanese patients after liver transplantation. Surg. Today 41, 1211–1218 (2011).

    Article  Google Scholar 

Download references

Author information

Authors and Affiliations


Corresponding authors

Correspondence to Gerard Said, Seden Grippon or Peter Kirkpatrick.

Ethics declarations

Competing interests

The authors declare no competing financial interests.

Rights and permissions

Reprints and Permissions

About this article

Cite this article

Said, G., Grippon, S. & Kirkpatrick, P. Tafamidis. Nat Rev Drug Discov 11, 185–186 (2012).

Download citation

  • Published:

  • Issue Date:

  • DOI:

This article is cited by


Quick links

Nature Briefing: Translational Research

Sign up for the Nature Briefing: Translational Research newsletter — top stories in biotechnology, drug discovery and pharma.

Get what matters in translational research, free to your inbox weekly. Sign up for Nature Briefing: Translational Research