Experimental treatments
for hATTR-PN

Diflunisal is an oral therapy thought to stabilize the TTR protein, which is expected to prevent the formation of toxic clumps and slow disease progression. Historically used off-label for some hATTR-PN cases, diflunisal is now approved in Europe (under the brand name Attrogy) for certain adults with the condition, though it remains unapproved for this indication in the U.S.

Nexiguran ziclumeran is an investigational gene-editing therapy in development for hATTR-PN that works to suppress TTR production and aggregate formation. It is given through a one-time infusion directly into the bloodstream.

Nucresiran is an experimental treatment being developed for hATTR-PN. Administered through under-the-skin injections, it is designed to lower TTR production, thereby preventing the formation of toxic aggregates.

Tafamidis meglumine is an oral TTR stabilizer therapy in development for hATTR-PN. It is approved for this indication under the brand name Vyndaqel in several regions outside the U.S.