Vutrisiran: a new drug in the treatment landscape of hereditary transthyretin amyloid polyneuropathy.
Expert Opin Drug Discov
; 19(4): 393-402, 2024 Apr.
Article
in En
| MEDLINE
| ID: mdl-38281068
ABSTRACT
INTRODUCTION:
Hereditary transthyretin (ATTRv) amyloidosis is a progressive, fatal disorder caused by mutations in the transthyretin (TTR) gene leading to deposition of the misfolded protein in amyloid fibrils. The main phenotypes are peripheral neuropathy (PN) and cardiomyopathy (CM). AREAS COVERED Gene silencing therapy, by dramatically reducing liver production of TTR, has transformed ATTRv-PN patient care in the last decade. In this drug discovery case history, the authors discuss the treatment history of ATTRv-PN and focus on the latest siRNA therapy vutrisiran. Vutrisiran is chemically enhanced and N-acetylgalactosamin-conjugated, allowing increased stability and specific liver delivery. HELIOS-A, a phase III, multicenter randomized study, tested vutrisiran in ATTRv-PN and showed significant improvement in neuropathy impairment, disability, quality of life (QoL), gait speed, and nutritional status. Tolerance was acceptable, no safety signals were raised. EXPERT OPINION Vutrisiran offers a new treatment option for patients with ATTRv-PN. Vutrisian's easier delivery and administration route, at a quarterly frequency, as well as the absence of premedication, are major improvements to reduce patients' disease burden and improve their QoL. Its place in the therapeutic strategy is to be determined, considering affordability.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Quality of Life
/
Amyloid Neuropathies
Type of study:
Clinical_trials
Aspects:
Patient_preference
Limits:
Humans
Language:
En
Journal:
Expert Opin Drug Discov
Year:
2024
Document type:
Article
Affiliation country: