Drug updated on 4/18/2024
| Dosage Form | Injection (subcutaneous; 189 mg/mL) |
| Drug Class | Aminolevulinate synthase 1-directed small interfering RNAs |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of adults with acute hepatic porphyria (AHP).
Summary
- Givosiran (Givlaari) is indicated for the treatment of adults with acute hepatic porphyria (AHP), a potentially fatal multiorgan dysfunction disorder. The primary mechanism involves the use of small interfering RNA molecules to mitigate the frequency and severity of acute attacks associated with AHP.
- One systematic review/meta-analysis included an analysis from 13 studies consisting of three clinical trials and ten review articles that collectively support Givosiran's efficacy in managing AHP's clinical manifestations.
- Givosiran has demonstrated significant effectiveness in reducing the rate of acute attacks in individuals with AHP, as well as in terminating recurrent attacks, marking it as a beneficial intervention for patients suffering from this severe condition.
- However, its safety profile is complex; side effects range from non-serious, such as fatigue and nausea, to more severe adverse effects like pain. There is also a lack of detailed information regarding Givosiran’s impact on renal and liver function - critical areas requiring further research, especially concerning its use in patients with pre-existing kidney or liver diseases.
- While there are no direct comparisons between Givosiran and other therapeutic options for AHP such as hemin or liver transplant within this analysis, it suggests that siRNA therapy through Givosiran represents a significant advancement offering a targeted approach addressing the underlying pathophysiology directly.
- Although specific population types or subgroup considerations were not explicitly mentioned within this analysis, it implies a need for further studies evaluating efficacy and safety among distinct patient subgroups, including those with varying degrees of kidney or liver function impairment.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Givlaari (givosiran) Prescribing Information. | 2023 | Alnylam Pharmaceuticals, Inc., Cambridge, MA |