Eplontersen for Hereditary Transthyretin Amyloidosis With Polyneuropathy
Phase III study (n=168, including 60 historical controls) found treatment with the antisense oligonucleotide eplontersen (45mg every 4 weeks) was associated with lower serum transthyretin concentration (−81.7% vs −11.2%), less neuropathy impairment, and better quality of life.
Source:
Journal of the American Medical Association
SPS commentary:
An accompanying editorial notes that eplontersen is a second-generation antisense oligonucleotide TTR silencer. It discusses the data so far for TTR gene silencers, and points out that while the new silencers do not improve on the ‘already remarkable’ results of patisiran, they have demonstrated an improved safety profile so far, with a longer administration interval and ease of use.