The first ever trials of a treatment for the progressive neurodegenerative disease TTR amyloid polyneuropathy (ATTR-PN), also known as familial amyloid polyneuropathy (FAP), has produced promising results.
ATTR-PN is an “orphan disease”, ie, one that has not been “adopted” by the pharmaceutical industry because it provides little financial incentive to make and market new medications.
In this case, FoldRx Pharmaceuticals was trialling its lead compound tafamidis (Fx-1006A) for a condition for which a liver transplant is otherwise the only available option.
Preliminary results show that tafamidis significantly halts disease progression, reduces the burden of the disease after 18 months and appears to be safe and well-tolerated.
FoldRx reports that no disease progression was observed in 60 per cent of patients after 18 months treatment compared with 38 per cent of those taking a placebo.
Placebo patients also experienced a significant deterioration in the Norfolk Quality Of Life measurement after 18 months. The company concludes that the trial resulted in “statistically significant and clinically meaningful improvements”.
Copyright Press Association 2009