Pegunigalsidase Alfa
Pegunigalsidase Alfa (ELFABRIO) is an enzyme replacement therapy designed to treat Fabry disease, a rare genetic lysosomal storage disorder caused by deficiency of the enzyme alpha-galactosidase A. It provides an exogenous source of the enzyme that is internalized by cells and transported to lysosomes, where it helps reduce the accumulation of globotriaosylceramide (Gb3) that causes organ damage in patients with Fabry disease.
Pegunigalsidase Alfa is an enzyme replacement therapy for adults with Fabry disease that provides exogenous alpha-galactosidase A to reduce Gb3 accumulation and manage disease symptoms.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- A16AB20
- RxNorm
- 2637446
- Generic
- Pegunigalsidase Alfa
- Class
- atc-a16
- Brands
- Elfabrio
- 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
- 500mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 250mg twice daily
- Maximum
- consult physician
- Frequency
- every 12 hours
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
- Usual dose
- 400mg daily
- Maximum
- consult physician
- Frequency
- once daily
- Instructions
- Follow physician instructions
- Duration limits
- as prescribed
- Monitoring
- standard
Overdose information
Special Populations
8 groupspregnancy
first trimesteravoid if possible
avoid use during pregnancy unless benefits outweigh risks
regular fetal monitoring and ultrasound assessments
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
second trimesteravoid if possible
discontinue therapy or switch to alternative management if pregnancy occurs
regular fetal monitoring
Use of this medication in this population requires careful consideration of risks and benefits.
pregnancy
third trimesteravoid if possible
consider discontinuing therapy near term
fetal monitoring and assessment of labor
Use of this medication in this population requires careful consideration of risks and benefits.
lactation
breastfeedingno adjustment needed
breastfeeding not recommended during treatment - discontinue breastfeeding or interrupt therapy
monitor infant for adverse effects
geriatric
elderlyno specific dose adjustment required
use standard dosing with close monitoring for adverse reactions
monitor renal and cardiac function regularly
Use with caution and monitor for adverse effects.
pediatric_age
adolescents 12 17weight-based dosing - 1mg/kg every 2 weeks
approved for patients 12 years and older - follow standard dosing protocol
monitor growth development and infusion-related reactions
renal_impairment
severe creatinine clearance 30no dose adjustment required in mild to moderate impairment
use with caution in severe renal impairment - monitor renal function closely
regular monitoring of renal function and proteinuria
Use with caution and monitor for adverse effects.
hepatic_impairment
mild to moderateno specific dose recommendation available
use with caution - limited clinical data in hepatic impairment
monitor liver function tests
Use with caution and monitor for adverse effects.
Statistics and clinical data
Drug class
Pegunigalsidase alfa is a PEGylated recombinant human alpha-galactosidase A enzyme
Indication
Long-term enzyme replacement therapy for adults with confirmed Fabry disease
Route of administration
Administered every 2 weeks via intravenous infusion
FDA approval year
FDA approved Pegunigalsidase alfa (Elfabrio) on May 6, 2022
ELFABRIO is indicated for long-term enzyme replacement therapy in adults with confirmed Fabry disease (alpha-galactosidase A deficiency). It is used to manage the disease by reducing Gb3 accumulation in various tissues and organs.
Pegunigalsidase Alfa is a recombinant form of alpha-galactosidase A. After intravenous infusion, the enzyme is internalized by patient cells through mannose-6-phosphate receptor-mediated endocytosis and transported to lysosomes, where it exerts its catalytic activity to break down accumulated Gb3 substrates.
As a protein-based therapeutic, ELFABRIO may cause infusion-related hypersensitivity reactions. Patients should be monitored during and after infusion. Cross-reactivity with anti-agalsidase antibodies may occur in patients previously treated with other enzyme replacement therapies. The safety and effectiveness in pediatric patients have not been established.
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
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
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