Vyndaqel: novel treatment for amyloid neuropathy

Pfizer has launched Vyndaqel (tafamidis) to delay peripheral neurologic impairment in patients with transthyretin familial amyloid polyneuropathy.

Vyndaqel (tafamidis) should be used in addition to standard care for transthyretin familial amyloid polyneuropathy.
Vyndaqel (tafamidis) should be used in addition to standard care for transthyretin familial amyloid polyneuropathy.

PHARMACOLOGY

Tafamidis is a novel specific stabiliser of transthyretin. Dissociation of the transthyretin tetramer to monomers is the rate-limiting step in the pathogenesis of transthyretin familial amyloid polyneuropathy. Tafamidis binds to the tetrameric form of transthyretin, inhibiting this dissociation and slowing disease progression.1

CLINICAL STUDIES

The safety and efficacy of tafamidis 20mg once daily were evaluated in a double-blind randomised, controlled study involving 128 patients with transthyretin familial amyloid polyneuropathy with the V30M mutation and primarily stage 1 disease.1

A pre-specified intention-to-treat analysis (n=125) after 18 months of treatment showed that more patients in the tafamidis group were responders on the Neuropathy Impairment Score of the Lower Limb (NIS-LL) scale than in the placebo group, although the difference was not significant (45.3% versus 29.5%, p=0.068).1

Analysis of secondary outcome measures showed that tafamidis treatment was associated with less deterioration of neurologic function and improved nutritional status compared with placebo.1

Safety profile

Tafamidis was generally well tolerated, with urinary tract infection and diarrhoea the most commonly reported adverse effects.1

References

  1. Vyndaqel Summary of Product Characteristics, December 2012

View Vyndaqel drug record

Further information:  Astellas

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