Pfizer hopes new analysis will persuade FDA to accept rare disease therapy

1 September 2017

US pharma giant Pfizer (NYSE: PFE) has published a new interim analysis of long-term data from four studies of its rare disease therapy Vyndaqel (tafamidis).

Published in the journal Amyloid, the analysis shows the drug is associated with delay in disease progression in patients with hereditary transthyretin amyloid polyneuropathy (TTR-FAP).

The analysis, which included patients with the most common TTR-FAP mutation, Val30Met, showed the drug was well tolerated, with no unexpected side effects.

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