GIVLAARI
GIVLAARI (givosiran sodium) is an aminolevulinate synthase 1-directed small interfering RNA indicated for the treatment of adults with acute hepatic porphyria (AHP). The medication is intended for adult patients to manage the manifestations of this metabolic disorder. It provides a targeted therapeutic approach by addressing the biological factors associated with disease attacks.
How GIVLAARI Works
Givosiran is a double-stranded small interfering RNA that triggers the degradation of aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference. This process reduces the elevated levels of liver ALAS1 mRNA, which in turn lowers the circulating levels of neurotoxic intermediates, specifically aminolevulinic acid (ALA) and porphobilinogen (PBG). By reducing these intermediates, the drug targets the primary factors associated with attacks and other manifestations of acute hepatic porphyria.
Details
- Status
- Prescription
- First Approved
- 2019-11-20
- Routes
- SUBCUTANEOUS
- Dosage Forms
- SOLUTION
GIVLAARI Approval History
What GIVLAARI Treats
1 indicationsGIVLAARI is approved for 1 conditions since its original approval in 2019. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute Hepatic Porphyria
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
GIVLAARI FDA Label Details
ProIndications & Usage
FDA Label (PDF)GIVLAARI is indicated for the treatment of adults with acute hepatic porphyria (AHP). GIVLAARI is an aminolevulinate synthase 1-directed small interfering RNA indicated for the treatment of adults with acute hepatic porphyria (AHP).
GIVLAARI Patents & Exclusivity
Patents (8 active)
Exclusivity
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.