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Vutrisiran · Amvuttra

Oligonucleotide (siRNA) · Gene silencing: transthyretin (TTR) · developed by Alnylam Pharms

Approved FDA, Jun 2022

ALNY Developer

1 Registered trials

What is Vutrisiran?

Vutrisiran is a double-stranded small interfering RNA conjugated to GalNAc that targets transthyretin (TTR) mRNA, both mutant and wild-type, for degradation through RNA interference. This reduces serum TTR protein and TTR deposits in tissues. It is studied in transthyretin amyloidosis with cardiomyopathy.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as: Amvuttra, ALN-TTRSC02
Drug type: Oligonucleotide (siRNA)
Targets: transthyretin (TTR)
Mechanism: Gene silencing: transthyretin (TTR)
Pathways: RNA interference
Therapy area: Metabolic
Developer: Alnylam Pharms Inc (ALNY)
FDA status: Approved as Amvuttra (June 13, 2022), NDA215515 · FDA label

✅ FDA-approved uses

Polyneuropathy of hereditary transthyretin-mediated amyloidosis
Cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis

From the indications section of the FDA label (version of Nov 6, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04153149	Phase 3	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy	Active not recruiting	ALNY

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

Other drugs acting on TTR

Inotersen FDA approved Patisiran FDA approved ALN-TTRSC04 Phase 1 NTLA-2001 Phase 3 Nucresiran Phase 3

More from Alnylam Pharms

Givosiran FDA approved Lumasiran FDA approved Patisiran FDA approved ALN-2232 Phase 1/2 ALN-4285 Phase 1 ALN-4324 Phase 2 ALN-4915 Phase 1 ALN-5288 Phase 1 ALN-6222 Phase 1 ALN-6400 Phase 1/2 ALN-AGT01 RVR Phase 2 ALN-BCAT Phase 1

Sources

Updated Oct 11, 2026. Something wrong or missing? Report it on the ALNY page.

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Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z