Elfabrio (pegunigalsidase alfa)
An approved treatment for Fabry Disease.
The same compound appears under different names depending on the context. Here is how to identify Pegunigalsidase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Pegunigalsidase alfa
- Brand name
- Elfabrio
- Development code
- PRX-102
- Drug class
- Enzyme replacement therapy (PEGylated)
- Manufacturer
- Chiesi Global Rare Diseases / Protalix BioTherapeutics
- How it's taken
- Given as an intravenous infusion every 2 weeks at 1 mg/kg.
A next-generation enzyme replacement therapy for adults with confirmed Fabry disease, with a chemically modified (PEGylated) enzyme designed for improved stability and longer action in the body compared to earlier ERT options.
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Where Pegunigalsidase alfa fits
Next-generation enzyme replacement for Fabry disease, given every 2 weeks like agalsidase beta (Fabrazyme). PEGylation extends how long the enzyme stays in the blood.
How Pegunigalsidase alfa works
Like Fabrazyme, Elfabrio provides the alpha-galactosidase A enzyme to break down accumulated GL-3. The difference is that PEGylation (attaching polyethylene glycol molecules) protects the enzyme from being broken down too quickly in the bloodstream, potentially allowing it to work longer and reach more affected tissues.
Mechanism: PEGylated enzyme replacement therapy with extended half-life for Fabry disease
Side effects and safety
Elfabrio has a boxed warning for serious allergic (hypersensitivity) reactions, including anaphylaxis, so it is given with emergency equipment available, and the infusion is stopped right away if a severe reaction happens. The most common side effects (15% or more) in the main trial were infusion-associated reactions, stuffy nose and sore throat (nasopharyngitis), headache, diarrhea, tiredness, nausea, back pain, pain in the arms or legs, and sinus infection. Your doctor may give antihistamines, fever reducers and/or steroids before infusions. The label also warns about a kidney inflammation called membranoproliferative glomerulonephritis, so kidney tests are monitored. Some patients develop antibodies to the drug.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Pegunigalsidase alfa
Given as an intravenous infusion every 2 weeks at 1 mg/kg. Made in plant cells. Infusions start at a healthcare facility; home infusion supervised by a healthcare provider may be considered once infusions are well tolerated.
Availability and cost
Only available as the brand-name product.
PEGylated enzyme replacement therapy with a longer half-life. In the US it is given every 2 weeks, the same schedule as agalsidase beta (Fabrazyme). Advanced biologic manufacturing with PEGylation technology for improved pharmacokinetics.
Help paying for Elfabrio
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Prescription Copay: eligible commercially insured patients may pay as little as $0 for Elfabrio. Government-funded plans are not eligible.
For: private insurance · source - Infusion cost help
Infusion Services Copay: may pay as little as $0 for infusion supplies and administration, including home infusion. Not for MA or RI.
For: private insurance · source - Insurance and case manager help
Patient Service Managers help with coverage, finding an infusion site, and home infusion eligibility.
The official page does not say who qualifies. Ask the program. · source
Good to know: Home infusion support requires a referral from your doctor. Uninsured patients may be eligible for financial assistance, but details are not given.
- From a charity · NORD RareCareFabry Disease Medical Assistance fundOpen
Pays for: Medical and medication costs.
The foundation says: “Accepting Applications” - From a charity · NORD RareCareFabry Disease Premium Copay Assistance fundOpen
Pays for: Insurance premiums and copays.
The foundation says: “Accepting Applications” - From a charity · TotalAssist (formerly PAN Foundation)Fabry Disease fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $9,000 per year. Requires health insurance (any kind).
- From a charity · Fabry Support & Information GroupFabry Assist fundApply directly
Pays for: One-time help with past-due Fabry-related medical bills, non-pharmacy medical items (cooling vests, compression stockings, hearing aids), Fabry testing and limited travel.
The foundation says: “Status not shown on page” - From a charity · The Assistance FundFabry Disease fundWaitlist
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “WAITLIST — Accepting Waitlist Patients. TAF is currently accepting requests to join the enrollment waitlist for this program. Waitlists a…”
How Fabry disease treatments compare
Three FDA-approved treatments are available for Fabry disease: two enzyme replacement therapies and one oral pharmacological chaperone. Your Fabry disease specialist (a geneticist, cardiologist, or nephrologist experienced with Fabry) will recommend a treatment based on your specific GLA mutation, disease severity, organ involvement, and personal preferences. Galafold is only an option if your mutation is classified as amenable.
3 FDA-approved fabry disease treatments are available: Fabrazyme (agalsidase beta, approved 2003); Galafold (migalastat, approved 2018); Elfabrio (pegunigalsidase alfa, approved 2023). Fabrazyme is typically used as standard first-line ert; 20+ years of clinical experience.
| Drug | How it works | How it’s given | How often | Where you get it | Typical use | FDA approved |
|---|---|---|---|---|---|---|
Fabrazyme agalsidase beta | Enzyme replacement — provides the alpha-galactosidase A enzyme your body can't make enough of, clearing GL-3 buildup from cells | Intravenous infusion (1 mg/kg) | Every 2 weeks | Infusion center or home infusion | Standard first-line ERT; 20+ years of clinical experience | 2003 |
Galafold migalastat | Pharmacological chaperone — stabilizes your body's own misfolded enzyme so it can fold correctly and function (only works for amenable GLA mutations) | Oral capsule (123 mg) on an empty stomach | Every other day (not on consecutive days) | Home (oral, no infusion needed) | Alternative to ERT for patients with amenable GLA mutations | 2018 |
Elfabrio pegunigalsidase alfa You are here | PEGylated enzyme replacement — same enzyme as Fabrazyme with PEG molecules attached for longer circulation and potentially better tissue penetration | Intravenous infusion (1 mg/kg) | Every 2 weeks | Infusion center or home infusion | Next-generation ERT; can shorten infusion time to 1.5 hours once established | 2023 |
This chart summarizes approved Fabry disease treatments to help you understand the landscape. It is not medical advice. Galafold only works for patients with specific amenable GLA gene mutations and must be confirmed by genetic testing before prescribing. Treatment decisions depend on your specific mutation, disease severity, organ involvement, and personal preferences. Always discuss options with your Fabry disease specialist, which may include a geneticist, cardiologist, or nephrologist depending on your primary organ involvement.
Clinical trial results
Demonstrated non-inferiority to agalsidase beta in the BALANCE trial. BRIDGE and BRIGHT studies showed sustained efficacy over 7.5 years of follow-up in 140+ patients.
Development history
Approved by FDA in May 2023 as the third enzyme replacement option for Fabry disease. Manufactured using a novel plant-cell-based production system by Protalix BioTherapeutics. Provides an alternative for patients who may not respond optimally to existing ERT.
Explore Fabry Disease trials
Other Fabry Disease treatments
Common questions about Pegunigalsidase alfa
▸What is Pegunigalsidase alfa (Elfabrio)?
A next-generation enzyme replacement therapy for adults with confirmed Fabry disease, with a chemically modified (PEGylated) enzyme designed for improved stability and longer action in the body compared to earlier ERT options.
▸How does Pegunigalsidase alfa work?
Like Fabrazyme, Elfabrio provides the alpha-galactosidase A enzyme to break down accumulated GL-3. The difference is that PEGylation (attaching polyethylene glycol molecules) protects the enzyme from being broken down too quickly in the bloodstream, potentially allowing it to work longer and reach more affected tissues.
▸What are the side effects of Pegunigalsidase alfa?
Elfabrio has a boxed warning for serious allergic (hypersensitivity) reactions, including anaphylaxis, so it is given with emergency equipment available, and the infusion is stopped right away if a severe reaction happens. The most common side effects (15% or more) in the main trial were infusion-associated reactions, stuffy nose and sore throat (nasopharyngitis), headache, diarrhea, tiredness, nausea, back pain, pain in the arms or legs, and sinus infection. Your doctor may give antihistamines, fever reducers and/or steroids before infusions. The label also warns about a kidney inflammation called membranoproliferative glomerulonephritis, so kidney tests are monitored. Some patients develop antibodies to the drug.
▸How is Pegunigalsidase alfa taken?
Given as an intravenous infusion every 2 weeks at 1 mg/kg. Made in plant cells. Infusions start at a healthcare facility; home infusion supervised by a healthcare provider may be considered once infusions are well tolerated.
▸Is Pegunigalsidase alfa FDA approved?
Yes, Pegunigalsidase alfa (Elfabrio) is FDA approved (2023) for the treatment of Fabry Disease.
▸What makes Elfabrio different from Fabrazyme?
Elfabrio is PEGylated (has polyethylene glycol molecules attached) which protects the enzyme from being broken down quickly, potentially allowing longer action in the body. It is also produced using plant cells rather than mammalian cells. Both are given as IV infusions every 2 weeks.
▸What did the BALANCE trial show?
The BALANCE trial demonstrated non-inferiority of pegunigalsidase alfa compared to agalsidase beta (Fabrazyme). The BRIDGE and BRIGHT studies showed sustained efficacy over 7.5 years of follow-up in over 140 patients.
▸How is Elfabrio manufactured differently?
Elfabrio is produced using a novel plant-cell-based production system by Protalix BioTherapeutics, using genetically modified tobacco plant cells (Bright Yellow 2, Nicotiana tabacum). This differs from the mammalian cell culture used for Fabrazyme and provides manufacturing diversity for the Fabry disease community.
▸Who should consider Elfabrio?
Elfabrio is approved for adults with confirmed Fabry disease who need enzyme replacement therapy; its safety and effectiveness in children have not been established. It provides a third ERT choice alongside Fabrazyme and was approved by the FDA in May 2023.
▸What patient support is available for Elfabrio?
Chiesi Total Care provides one-stop patient support with copay assistance, insurance navigation, and infusion support for Elfabrio patients.
▸Can patients switch from Fabrazyme to Elfabrio?
Yes, clinical trials included patients who switched from other enzyme replacement therapies to pegunigalsidase alfa. Your doctor can evaluate whether switching is appropriate based on your individual response and clinical situation.