Lumasiran
Lumasiran is an RNA interference (RNAi) therapeutic agent designed to treat primary hyperoxaluria type 1 (PH1), a rare genetic disorder characterized by excessive oxalate production. By reducing the glycolate oxidase enzyme in liver cells, lumasiran decreases oxalate synthesis, thereby preventing the formation of kidney stones and renal damage associated with this condition.
Lumasiran is an RNAi therapy that reduces oxalate production in the liver for patients with primary hyperoxaluria type 1.
Key facts at a glance
- ATC
- A16AX18
- RxNorm
- 2467140
- Generic
- Lumasiran
- Class
- atc-a16
- Brands
- Oxlumo
- 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
- 150mg once monthly (after loading dose)
- Maximum
- consult physician
- Frequency
- once monthly (loading: 150mg daily x 3 doses)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 3mg/kg once monthly (after loading dose)
- Maximum
- consult physician
- Frequency
- once monthly (loading: 3mg/kg daily x 3 doses)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 3mg/kg once monthly or 150mg fixed dose (after loading dose)
- Maximum
- consult physician
- Frequency
- once monthly (loading: 3mg/kg daily x 3 doses or 150mg daily x 3 doses)
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
11 groupspregnancy
first trimesteravoid if possible
use only if benefits outweigh potential risks to fetus
regular fetal monitoring
Use with caution and monitor for adverse effects.
pregnancy
second trimesteravoid if possible
continue only if benefits outweigh risks
regular fetal monitoring and ultrasound
Use with caution and monitor for adverse effects.
pregnancy
third trimesteravoid if possible
consider discontinuing near term if clinically appropriate
fetal monitoring and assessment of neonatal outcomes
Use with caution and monitor for adverse effects.
lactation
breastfeedingno adjustment
discontinue breastfeeding or discontinue drug based on risk-benefit assessment
monitor infant for adverse effects
pediatric_age
infantsweight-based dosing
approved for use in pediatric patients including infants
monitor urinary oxalate levels and renal function
pediatric_age
children 2 to 11weight-based dosing
approved for use in pediatric patients
regular monitoring of urinary oxalate and liver function
geriatric
elderlyno specific adjustment
use with caution due to potential comorbidities
monitor renal and hepatic function
Use with caution and monitor for adverse effects.
renal_impairment
moderateno specific adjustment
use with caution; limited data in patients with eGFR 30-59
close monitoring of renal function and oxalate levels
Use with caution and monitor for adverse effects.
renal_impairment
severeuse with extreme caution
not recommended in patients with severe renal impairment or ESRD
intensive monitoring of renal function and oxalate levels
Use with caution and monitor for adverse effects.
hepatic_impairment
mildno specific adjustment
use with caution in mild hepatic impairment
monitor liver function tests
Use with caution and monitor for adverse effects.
hepatic_impairment
moderate to severeuse with caution
use with caution; not studied in moderate to severe hepatic impairment
monitor liver function and clinical response
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Lumasiran is an siRNA (small interfering RNA) therapeutic
Indication
Therapeutic indication for Oxlumo
FDA approval year
FDA approval date for Oxlumo
Mechanism of action
Primary pharmacological mechanism
Lumasiran is indicated for the treatment of primary hyperoxaluria type 1 (PH1) in both adults and pediatric patients. PH1 is a rare autosomal recessive disorder caused by deficiency of the enzyme alanine-glyoxylate aminotransferase (AGT), leading to overproduction of oxalate, which can cause recurrent kidney stones, nephrocalcinosis, and systemic oxalosis.
Lumasiran utilizes small interfering RNA (siRNA) technology to target and degrade hydroxyacid oxidase 1 (HAO1) messenger RNA in hepatocytes. This reduces production of the glycolate oxidase enzyme, which in turn decreases the conversion of glycolate to glyoxylate. With less glyoxylate available as a substrate, oxalate synthesis is significantly reduced, lowering urinary and plasma oxalate levels.
Lumasiran is administered by subcutaneous injection, typically with loading doses followed by maintenance doses every three months. Common adverse reactions include injection site reactions and abdominal pain. Patients should be monitored for reductions in plasma oxalate levels and may require concurrent pyridoxine (vitamin B6) supplementation in some cases. This medication should be used under the supervision of physicians experienced in managing metabolic disorders.
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