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 not distributed through mainland China's retail pharmacy network. Uwell Cloud can help arrange a purchase channel for this medicine, sourced and dispatched from Hong Kong; clinical administration support in China can be coordinated on request. Availability and eligibility for any specific patient are determined by clinical review.

Regulatory Approvals

• FDA — Approved (2022) — Sources
• EMA — Approved (2022) — Sources

Pivotal Clinical Evidence

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

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