8 approved drugs

Approved siRNA therapeutics

All eight approved small interfering RNA therapeutics — patisiran, givosiran, lumasiran, inclisiran, vutrisiran, nedosiran, fitusiran and plozasiran — with target, indication, delivery chemistry and links to every FDA and EMA source document in the corpus.

The class at a glance

Approved siRNA therapeutics with molecular target, indication, delivery route and regulatory coverage
DrugTargetIndicationDeliveryDocuments
Patisiran (Onpattro)TTRhATTR amyloidosis (polyneuropathy)LNP, IV5 FDA + EMA
Givosiran (Givlaari)ALAS1Acute hepatic porphyriaGalNAc, SC4 FDA + EMA
Lumasiran (Oxlumo)HAO1Primary hyperoxaluria type 1GalNAc, SC4 FDA + EMA
Inclisiran (Leqvio)PCSK9Hypercholesterolemia / ASCVDGalNAc, SC5 FDA + EMA
Vutrisiran (Amvuttra)TTRhATTR polyneuropathy; ATTR-CMGalNAc, SC5 FDA + EMA
Nedosiran (Rivfloza)LDHAPrimary hyperoxaluria type 1GalNAc, SC2 FDA
Fitusiran (Qfitlia)SERPINC1 (antithrombin)Hemophilia A/BGalNAc, SC2 FDA
Plozasiran (Redemplo (EU))APOC3Familial chylomicronemia syndromeGalNAc, SC4 FDA + EMA

What sits behind each page

Each drug page lists the primary regulatory documents held in the corpus and links straight to them at FDA and EMA. Pharmacological detail — dosing, exposure, safety findings — is answered from those documents in the chat, with a citation on every claim, rather than restated here. The corpus holds 20 FDA documents, 12 EMA documents and 79 publications in total.