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Givlaari (GIVOSIRAN)
Givlaari works by binding to and silencing the messenger RNA (mRNA) that produces the enzyme aminolevulinate synthase 1 (ALAS1), which is overproduced in patients with hepatic porphyria.
Givlaari (givosiran) is a small molecule developed by Alnylam Pharmaceuticals, targeting aminolevulinate synthase 1 (ALAS1) mRNA to treat hepatic porphyria. It is an aminolevulinate synthase 1-directed RNA interaction drug, approved by the FDA in 2019. Givlaari is a patented medication with no generic manufacturers available. Key safety considerations include the potential for infusion-related reactions and liver enzyme elevations. It is used to treat a group of rare genetic disorders known as hepatic porphyrias.
At a glance
| Generic name | GIVOSIRAN |
|---|---|
| Sponsor | Alnylam Pharmaceuticals |
| Drug class | Aminolevulinate Synthase 1-directed RNA Interaction |
| Target | aminolevulinate synthase1 (ALAS1) mRNA |
| Modality | Oligonucleotide |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2019 |
| Annual revenue | 400 |
Mechanism of action
Givosiran is double-stranded small interfering RNA that causes degradation of aminolevulinate synthase (ALAS1) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and porphobilinogen (PBG), factors associated with attacks and other disease manifestations of AHP.
Approved indications
- Hepatic porphyria
Common side effects
- nausea
- injection site reactions
- rash
- serum creatinine increase
- transaminase elevations
- fatigue
Key clinical trials
- ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
- Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
- ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP) (PHASE3)
- A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP) (PHASE1,PHASE2)
- A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP) (PHASE1)
- A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP) (PHASE1)
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
| SEC EDGAR | Revenue + earnings |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Givlaari CI brief — competitive landscape report
- Givlaari updates RSS · CI watch RSS
- Alnylam Pharmaceuticals portfolio CI