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Givosiran · Givlaari

Oligonucleotide (siRNA) · Gene silencing: aminolevulinate synthase 1 (ALAS1) · developed by Alnylam Pharms

Approved FDA, Nov 2019

ALNY Developer

1 Registered trials

What is Givosiran?

Givosiran is a double-stranded small interfering RNA conjugated to GalNAc, which is taken up by hepatocytes through the asialoglycoprotein receptor. It degrades ALAS1 mRNA through RNA interference, reducing hepatic ALAS1 production and the levels of neurotoxic intermediates ALA and porphobilinogen. It is used in the treatment of acute hepatic porphyria.

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

At a glance

Also known as: Givlaari, ALN-AS1
Drug type: Oligonucleotide (siRNA)
Targets: aminolevulinate synthase 1 (ALAS1)
Mechanism: Gene silencing: aminolevulinate synthase 1 (ALAS1)
Pathways: heme biosynthesis, ALA and PBG production
Therapy area: Gastroenterology
Developer: Alnylam Pharms Inc (ALNY)
FDA status: Approved as Givlaari (November 20, 2019), NDA212194 · FDA label

✅ FDA-approved uses

Acute hepatic porphyria

From the indications section of the FDA label (version of Sep 10, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trial	Phase	Condition	Status	Sponsor
NCT04056481	N/A	Acute Hepatic Porphyria	Approved for marketing	ALNY

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

Conditions studied

Acute Hepatic Porphyria

More from Alnylam Pharms

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 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