Amvuttra
E446848
Amvuttra is an RNA interference (RNAi) therapeutic used to treat hereditary transthyretin-mediated (hATTR) amyloidosis by reducing the production of transthyretin protein.
Statements (44)
| Predicate | Object |
|---|---|
| instanceOf |
RNA interference therapeutic
ⓘ
drug ⓘ small interfering RNA ⓘ transthyretin-directed therapy ⓘ |
| approvalYear | 2022 ⓘ |
| belongsToPlatform | RNAi therapeutics platform ⓘ |
| countryApprovedIn |
European Union
NERFINISHED
ⓘ
United States NERFINISHED ⓘ |
| developer | Alnylam Pharmaceuticals NERFINISHED ⓘ |
| dosingFrequency | once every three months ⓘ |
| effect | reduces production of transthyretin protein ⓘ |
| hasATCClass | N07XX ⓘ |
| hasDosingInterval | 12 weeks ⓘ |
| hasDrugClass |
RNAi therapeutic
ⓘ
gene silencing agent ⓘ transthyretin synthesis inhibitor ⓘ |
| hasEffectOn | polyneuropathy progression in hATTR amyloidosis ⓘ |
| hasFormulation | solution for injection ⓘ |
| hasGenericName | vutrisiran ⓘ |
| hasInternationalNonproprietaryName | vutrisiran NERFINISHED ⓘ |
| hasMolecularTarget | transthyretin NERFINISHED ⓘ |
| hasPharmacologicalAction | gene expression inhibitor ⓘ |
| hasRegulatoryStatus |
EMA approved
ⓘ
FDA approved ⓘ |
| indication | treatment of adults with hereditary transthyretin-mediated amyloidosis ⓘ |
| isAdministered | subcutaneously ⓘ |
| isAdministeredBy | healthcare professional ⓘ |
| isFirstInClass | subcutaneous RNAi therapeutic for hATTR polyneuropathy ⓘ |
| isIndicatedFor | adult patients ⓘ |
| isLongActing | true ⓘ |
| isNotIndicatedFor | pediatric patients ⓘ |
| isProprietaryNameOf | vutrisiran NERFINISHED ⓘ |
| isUsedFor | disease-modifying treatment of hATTR amyloidosis ⓘ |
| marketingAuthorizationHolder | Alnylam Pharmaceuticals NERFINISHED ⓘ |
| mechanismOfAction | RNA interference–mediated degradation of transthyretin mRNA ⓘ |
| reduces |
amyloidogenic transthyretin variants
ⓘ
serum transthyretin levels ⓘ |
| routeOfAdministration | subcutaneous injection ⓘ |
| targets | transthyretin mRNA ⓘ |
| therapeuticArea |
amyloidosis
ⓘ
neurology ⓘ rare diseases ⓘ |
| treats |
hereditary transthyretin-mediated amyloidosis
ⓘ
polyneuropathy of hereditary transthyretin-mediated amyloidosis ⓘ |
Referenced by (1)
Full triples — surface form annotated when it differs from this entity's canonical label.