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givosiran
- Development stage
- Approved
- Lead developer
- Alnylam Pharmaceuticals
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Subcutaneous
01
Overview
Givosiran is a double-stranded small interfering RNA (siRNA) therapeutic designed to treat acute hepatic porphyria (AHP), a group of rare, inherited metabolic disorders characterized by potentially life-threatening attacks and chronic symptoms. It works by specifically targeting and degrading aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference, leading to reduced production of the ALAS1 enzyme. This results in decreased levels of neurotoxic heme intermediates such as delta-aminolevulinic acid (ALA) and porphobilinogen (PBG), which are associated with AHP attacks and other disease manifestations. Givosiran is administered as a once-monthly subcutaneous injection. The drug was developed by Alnylam Pharmaceuticals.
- Brand names
- Givlaari
- Other names
- givosiran sodium
02
Targets
ALAS1 (5-aminolevulinate synthase 1)ASGPR (Asialoglycoprotein Receptor 1)
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