New Clinical Research Findings for ONPATTRO® (patisiran) at EU-ATTR Amyloidosis Meeting

New Clinical Research Findings for ONPATTRO® (patisiran) at EU-ATTR Amyloidosis Meeting

We presented results from a proteome-wide biomarker analysis of samples from the APOLLO Phase 3 study of ONPATTRO® (patisiran), as well as results from an analysis of the UK Biobank on clinical outcomes and medical history of individuals with the non-pathogenic transthyretin (TTR) ‘stabilizing’ T119M variant, at the Second European Meeting of ATTR Amyloidosis (EU-ATTR) for Doctors and Patients, held September 2-3 in Berlin, Germany.

Read our press release

Ticau et al. – “Neurofilament Light Chain (NfL) as a Potential Biomarker in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis”

Parker et al. – “The Transthyretin Stabilizing Mutation (T119M) Is Not Associated with Extended Lifespan or Protection Against Vascular Diseases: Analysis of the UK Biobank Cohort”

Data from the APOLLO biomarker analysis support further evaluation of neurofilament light chain (NfL) as a biomarker for hATTR amyloidosis that may facilitate earlier diagnosis of polyneuropathy and enable monitoring of disease progression and/or reversal of disease over time. Moreover, upon further evaluation, NfL may also offer an easy and convenient blood test to detect polyneuropathy in patients with mutations that predominantly cause cardiomyopathy (e.g., V122I), but where underlying nerve damage does often occur and can be overlooked.

The UK Biobank analysis showed that carriers of the TTR T119M variant were not protected against vascular, cardiovascular, or cerebrovascular disease, or death. Furthermore, no difference was seen between T119M carriers and non-carriers in their time to death following a diagnosis of vascular disease.


Receive news and updates on the work at Alnylam that affects you most.