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Patisiran · Onpattro, ALN-18328

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

Approved FDA, Aug 2018

ALNY Developer

2 Registered trials

What is Patisiran?

Patisiran is a double-stranded small interfering RNA that binds transthyretin (TTR) mRNA and causes its degradation through RNA interference, reducing serum TTR protein and TTR deposits in tissues. It is delivered by intravenous infusion and is studied in transthyretin-mediated amyloidosis, including amyloid polyneuropathy and cardiomyopathy.

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

At a glance

Also known as: Onpattro, ALN-18328, SAR-438037, ALN-TTR02
Drug type: Oligonucleotide (siRNA)
Targets: transthyretin (TTR)
Mechanism: Gene silencing: transthyretin (TTR)
Pathways: RNA interference
Therapy area: Neurology
Developer: Alnylam Pharms Inc (ALNY)
FDA status: Approved as Onpattro (August 10, 2018), NDA210922 · FDA label
UNII: 50FKX8CB2Y

✅ FDA-approved uses

Polyneuropathy of hereditary transthyretin-mediated amyloidosis

From the indications section of the FDA label (version of Sep 8, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT03997383	Phase 3	Transthyretin Amyloidosis (ATTR) With Cardiomyopathy	Completed	ALNY
NCT02939820	N/A	TTR-mediated Amyloidosis	Approved for marketing	ALNY

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

Conditions studied

Amyloid NeuropathiesAmyloid Neuropathies, FamilialAmyloidosis, HereditaryAmyloidosis, Hereditary, Transthyretin-RelatedFamilial Amyloid PolyneuropathiesTransthyretin Amyloidosis (ATTR) With CardiomyopathyTTR-mediated Amyloidosis

Other drugs acting on TTR

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

More from Alnylam Pharms

Givosiran FDA approved Lumasiran FDA approved Vutrisiran 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