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Cemdisiran

Oligonucleotide (siRNA) · Gene silencing: Complement component 5 (C5) (C5) · developed by ALNYLAM PHARMACEUTICALS

Phase 2 Most advanced trial (none active)

ALNY Developer

1 Registered trials

What is Cemdisiran?

Cemdisiran is a small-interfering RNA conjugated to a GalNAc ligand that is taken up by hepatocytes via the asialoglycoprotein receptor. Inside the cell it binds C5 mRNA, reducing C5 protein production and lowering plasma C5 to limit complement activation. It is studied in IgA nephropathy and paroxysmal nocturnal hemoglobinuria.

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

At a glance

Also known as: ALN-CC5
Drug type: Oligonucleotide (siRNA)
Targets: Complement component 5 (C5) (C5)
Mechanism: Gene silencing: Complement component 5 (C5) (C5)
Pathways: complement pathway, complement-mediated hemolysis
Therapy area: Renal & urology
Developer: ALNYLAM PHARMACEUTICALS (ALNY)
FDA status: Investigational, not approved in the US. Most advanced trial: Phase 2
UNII: S66Z65E10T

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT03841448	Phase 2	IgA Nephropathy (IgAN)	Terminated	ALNY

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

Conditions studied

Berger DiseaseGlomerulonephritis, IgAIgA Nephropathy (IgAN)

Other drugs acting on C5

Nomacopan Phase 3 Tanruprubart Phase 3

More from ALNYLAM PHARMACEUTICALS

Givosiran FDA approved Lumasiran FDA approved Patisiran 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

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