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Givosiran: A Review in Acute Hepatic Porphyria.
- Source :
-
Drugs [Drugs] 2021 May; Vol. 81 (7), pp. 841-848. Date of Electronic Publication: 2021 Apr 19. - Publication Year :
- 2021
-
Abstract
- Givosiran (Givlaari <superscript>®</superscript> ) is an δ-aminolevulinic acid synthase 1 (ALAS1)-directed small interfering RNA (siRNA) approved for the treatment of acute hepatic porphyria (AHP). In the phase 3 ENVISION trial, givosiran significantly reduced the annualized rate of composite porphyria attacks (i.e. attacks requiring hospitalization, urgent healthcare visit or intravenous hemin administration at home) compared with placebo in patients with recurrent acute intermittent porphyria (the most common type of AHP) attacks. Givosiran also improved several other outcomes, including hemin use and pain (the cardinal symptom of AHP). While generally well tolerated with an acceptable safety profile, the drug may increase the risk of hepatic and kidney adverse events. Givosiran offers the convenience of once-monthly subcutaneous administration. Available evidence indicates that givosiran is an important newer therapeutic option for patients with AHP and severe recurrent attacks.
- Subjects :
- 5-Aminolevulinate Synthetase antagonists & inhibitors
Acetylgalactosamine adverse effects
Acetylgalactosamine pharmacokinetics
Acetylgalactosamine pharmacology
Acetylgalactosamine therapeutic use
Acute Kidney Injury chemically induced
Chemical and Drug Induced Liver Injury
Drug Interactions
Hemin administration & dosage
Hospitalization
Humans
Pain drug therapy
Pain etiology
Porphyria, Acute Intermittent complications
Porphyrias, Hepatic complications
Pyrrolidines adverse effects
Pyrrolidines pharmacokinetics
RNA, Small Interfering
Randomized Controlled Trials as Topic
Severity of Illness Index
Acetylgalactosamine analogs & derivatives
Porphobilinogen Synthase deficiency
Porphyria, Acute Intermittent drug therapy
Porphyrias, Hepatic drug therapy
Pyrrolidines pharmacology
Pyrrolidines therapeutic use
Subjects
Details
- Language :
- English
- ISSN :
- 1179-1950
- Volume :
- 81
- Issue :
- 7
- Database :
- MEDLINE
- Journal :
- Drugs
- Publication Type :
- Academic Journal
- Accession number :
- 33871817
- Full Text :
- https://doi.org/10.1007/s40265-021-01511-3