Skip to main content

Advertisement

SpringerLink
Log in

Tafamidis

  • Fresh from the Pipeline
  • Published:

From Nature Reviews Drug Discovery

View current issue Sign up to alerts

Abstract

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, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Figure 1: Tafamidis.

References

  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

  1. Gerard Said is at the Department of Neurology, Centre Hospitalier, Universitaire Pitié-Salpêtrière, 75651 Paris Cedex 13, France.,

    Gerard Said

  2. Seden Grippon is at IMS Health, 7 Harewood Avenue, London NW1 6JB, UK.,

    Seden Grippon

  3. Peter Kirkpatrick is at Nature Reviews Drug Discovery. ,

    Peter Kirkpatrick

Authors
  1. Gerard Said
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Seden Grippon
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Peter Kirkpatrick
    View author publications

    You can also search for this author in PubMed Google Scholar

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). https://doi.org/10.1038/nrd3675

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/nrd3675

  • Springer Nature Limited

This article is cited by

Search

Navigation