Givosiran
Givosiran is an RNA interference therapeutic used to treat acute hepatic porphyria, a rare genetic disorder characterized by dangerous accumulations of neurotoxic porphyrin precursors that can cause severe abdominal pain, neurological complications, and potentially life-threatening attacks. By reducing liver ALAS1 enzyme levels through targeted mRNA degradation, givosiran decreases the production of these toxic intermediates and helps prevent acute porphyria attacks. It is specifically indicated for patients with acute intermittent porphyria and related subtypes of acute hepatic porphyria who experience recurrent attacks.
Givosiran is an siRNA therapy that treats acute hepatic porphyria by reducing liver ALAS1 enzyme to prevent accumulation of neurotoxic porphyrin precursors and acute attacks.
Key facts at a glance
- ATC
- A16AX16
- RxNorm
- 2265712
- Generic
- Givosiran
- Class
- atc-a16
- Brands
- Givlaari
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Dosage and administration
- Usual dose
- 2.5 mg/kg once monthly
- Maximum
- consult physician
- Frequency
- once monthly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2.5 mg/kg once monthly
- Maximum
- consult physician
- Frequency
- once monthly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 2.5 mg/kg once monthly
- Maximum
- consult physician
- Frequency
- once monthly
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
all trimestersavoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring recommended
Use with caution and monitor for adverse effects.
lactation
breastfeedingno adjustment
discontinue breastfeeding or discontinue drug; decision should be made considering importance of therapy to mother
monitor infant for adverse effects
geriatric
elderly 65 years and olderno adjustment
no dose adjustment required based on age alone; monitor renal and hepatic function
periodic monitoring of renal and hepatic function
pediatric_age
children adolescentsnot established
safety and efficacy not established in patients under 18 years
not applicable
renal_impairment
mild to moderateno adjustment
no dose adjustment needed for mild to moderate renal impairment
routine monitoring recommended
renal_impairment
severeuse with caution
use with caution; limited data available for severe renal impairment
close monitoring of renal function recommended
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno adjustment
no dose adjustment needed for mild to moderate hepatic impairment
routine monitoring of liver function tests
hepatic_impairment
severeuse with caution
use with caution; not studied in severe hepatic impairment
close monitoring of liver function tests recommended
Use with caution and monitor for adverse effects.
Statistics and clinical data
FDA approval year
FDA approval date for Givlaari
Drug class
Classification as RNA interference therapeutic
Molecular target
Primary molecular target of givosiran
Indication
Therapeutic indication for treatment
Treatment of acute hepatic porphyria (AHP) in adults, including acute intermittent porphyria (AIP), hereditary coproporphyria (HCP), and variegate porphyria (VP) in patients experiencing recurrent attacks.
Double-stranded small interfering RNA (siRNA) that selectively degrades aminolevulinate synthase 1 (ALAS1) mRNA in hepatocytes through RNA interference, reducing liver ALAS1 enzyme levels and subsequently lowering circulating levels of neurotoxic porphyrin precursors aminolevulinic acid (ALA) and porphobilinogen (PBG).
Administered as a subcutaneous injection, typically once monthly. Requires monitoring of liver function tests, urinary ALA, and porphobilinogen levels. Common adverse effects include injection site reactions, nausea, fatigue, and transient increases in liver transaminases. Should be used with caution in patients with moderate or severe hepatic impairment.
Contraindications
Conditions and patient states where this medication should be avoided or used with caution.
Do not use (1)
Known allergy to this medication or its components
Use caution (1)
Consult healthcare provider for benefits vs risks assessment
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy