AMVUTTRA vutrisiran

Drug Class

Transthyretin-directed double-stranded small interfering RNA (siRNA)

Mechanism

Vutrisiran targets transthyretin (TTR) messenger RNA to cause its degradation, reducing the production of mutant and wild-type TTR protein in the liver.

Approved Indication

Treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.

What This Means For Patients

Amvuttra is an RNA interference (RNAi) therapeutic administered subcutaneously once every three months for adults with hATTR amyloidosis polyneuropathy. It works by silencing the gene responsible for producing the abnormal transthyretin protein. It is available under Hainan Boao Lecheng's special-import drug policy.

Regulatory Approvals

• FDA — approved (2022) — label
• EMA — approved (2022) — source

Pivotal Clinical Evidence

• HELIOS-A — Adams et al., Amyloid 2023 (NCT03759379) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/215515s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/amvuttra
• https://www.alnylam.com/news-release/alnylam-announces-fda-approval-of-amvuttra-vutrisiran-for-the-treatment-of-the-polyneuropathy-of-hereditary-transthyretin-mediated-amyloidosis-in-adults