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The mechanism of ATTR4,5

Amyloidogenic TTR cascade

Secreted as a tetramer
The tetramer breaks into monomers
Amyloid fibril
Misfolded monomers aggregate into amyloid fibrils
Deposition of
Amyloid fibrils accumulate in different parts of the body, such as the nerves

Vyndaqel® is a selective stabilizer of TTR4,5

* As measured by a TTR stabilization assay.
† Study design: In the 1.5-year, randomized, double-blind, placebo-controlled pivotal study, Val30Met patients received Vyndaqel® 20mg once daily or placebo. Coprimary endpoints: Neuropathy Impairment score of The Lower Limb (NIS-LL) responder analysis (change of less than 2 points on NIS-LL) and treatment group difference in the mean change from baseline in Norfolk Quality of Life-Diabetic Neuropathy total score ((Norfolk QOL-DN) in ITT population (n = 125) and EE (n=87) populations. Secondary endpoints: changes in neurological function, small and large nerve fiber function, and nutritional status.8
ⱡ Study design: A 1-year, open-label, single-arm study. Non-Val30Met patients received Vyndaqel® 20mg once daily. Primary endpoint TTR stabilization at week 6 (n=19). Secondary endpoints: TTR stabilization at month 6 (n=18) and month 12 (n=17).9