There is more than one approach to Fabry treatment

Discover how options can support your patients’ journey

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For years, patients with Fabry disease had limited treatment options and only one enzyme replacement therapy (ERT) available.2,3

However, with Elfabrio’s approval in May 2023, the first option is no longer the only option.1-3

Now in Fabry disease treatment,
you have a choice.1,3

Elfabrio is the only PEGylated ERT for adults with Fabry disease. This PEGylation process extends the treatment’s half-life,* resulting in Elfabrio staying in the body between doses and blood levels increasing over time with repeat administration.1,3,4

Patients on Elfabrio showed†:

•

Long-term renal stability after more than 5 years in
treatment-naive patients5‡

•

Low rates of treatment-emergent adverse events (TEAEs)5,6§

•

Low rates of infusion-related reactions (IRRs) at 24 hours8,9¶

•

Low immunogenicity5-7#**

Whether your patient is newly diagnosed or is looking to switch their current treatment, you can explore different options in Fabry care. With more than one option available, you have the flexibility to choose the treatment that most appropriately suits your patients’ needs. With Elfabrio, you can offer your patients a treatment that is long lasting,* maintains renal function, and is generally well tolerated.1,3-9

Elfabrio has an initial half-life of 78.9 ± 10.3 hours. Clinical studies have not established that pharmacological characteristics, including half-life, result in superior efficacy or safety based on clinically relevant endpoints. Infusions are every 2 weeks.1

^†

Based on a long-term trial in treatment-naïve patients (NAÏVE) and a 2-year head-to-head trial vs agalsidase beta in switch patients (BALANCE). BALANCE provided confirmatory evidence in the FDA’s approval of Elfabrio. The data discussed are not intended to establish noninferiority or superiority to any other marketed drug product on the basis of safety or efficacy. In the Elfabrio Prescribing Information, NAÏVE is referred to as Trial 1 and BALANCE as Trial 2.1,5,6

^‡

eGFR mean slope at 5 years was -1.6 mL/min/1.73 m²/year (n=15 at baseline; n=10 at 60 months).5

^§

In a head-to-head trial, Elfabrio-treated patients demonstrated a 3.6x lower TEAE rate per 100 years of exposure vs agalsidase beta. Among 52 Elfabrio-treated patients, there were 42 TEAEs. Among 25 patients treated with agalsidase beta, there were 76 TEAEs.6

^¶

In an integrated safety analysis of both treatment-naïve and treatment-experienced patients, IRR rates at 24 hours demonstrated that 28.8% of Elfabrio-treated patients (32/111) experienced an IRR (71 events).9

The effect of IgG ADAs on the effectiveness of Elfabrio has not been fully characterized. The detection of antibody formation is highly dependent on the sensitivity and specificity of the assay. Go to hcp.elfabrio.com to see the low rates of ADA formation.

In that same integrated analysis, 79% of Elfabrio-treated patients (64/81) who were ADA negative at baseline remained ADA negative during treatment.7

Explore your options.

ADAs, anti-drug antibodies; eGFR, estimated glomerular filtration rate; IgG, immunoglobulin G; PEG, polyethylene glycol.

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Indication

Elfabrio® (pegunigalsidase alfa-iwxj) is indicated for the treatment of adults with confirmed Fabry disease.

Important Safety Information

WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS

Patients treated with Elfabrio have experienced hypersensitivity reactions, including anaphylaxis. Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available during Elfabrio administration. If a severe hypersensitivity reaction (eg, anaphylaxis) occurs, discontinue Elfabrio immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reaction, a desensitization procedure to Elfabrio may be considered.

Prior to Elfabrio administration, consider pretreating with antihistamines, antipyretics, and/or corticosteroids. Inform patients and caregivers of the signs and symptoms of hypersensitivity reactions and infusion-associated reactions (IARs), and instruct them to seek medical care immediately if such symptoms occur.

•

If a severe hypersensitivity reaction (including anaphylaxis) or severe IAR occurs, immediately discontinue Elfabrio administration and initiate appropriate medical treatment.

•

If a mild to moderate hypersensitivity reaction or IAR occurs, consider slowing the infusion rate or temporarily withholding the dose.

In clinical trials, 20 (14%) Elfabrio-treated patients experienced hypersensitivity reactions. Four Elfabrio-treated patients (3%) experienced anaphylaxis reactions that occurred within 5 to 40 minutes of the start of the initial infusion. The signs and symptoms of hypersensitivity reactions and anaphylaxis included headache, nausea, vomiting, throat tightness, facial and oral edema, truncal rash, tachycardia, hypotension, rigors, urticaria, intense pruritus, moderate upper airway obstructions, macroglossia, and mild lip edema.

In clinical trials, 41 (29%) Elfabrio-treated patients experienced one or more infusion-associated reactions, including hypersensitivity, nausea, chills, pruritus, rash, chest pain, dizziness, vomiting, asthenia, pain, sneezing, dyspnea, nasal congestion, throat irritation, abdominal pain, erythema, diarrhea, burning sensation, neuralgia, headache, paresthesia, tremor, agitation, increased body temperature, flushing, bradycardia, myalgia, hypertension, and hypotension.

A case of membranoproliferative glomerulonephritis with immune depositions in the kidney was reported during clinical trials. Monitor serum creatinine and urinary protein-to-creatinine ratio. If glomerulonephritis is suspected, discontinue treatment until a diagnostic evaluation can be conducted.

When switching to Elfabrio from a prior enzyme replacement therapy, the risk of hypersensitivity reactions and infusion-associated reactions may be increased in certain patients with pre-existing anti-drug antibodies (ADAs). Consider monitoring IgG and IgE ADAs and clinical or pharmacodynamic response (eg, plasma lyso-Gb3 levels).

The most common adverse reactions (≥15%) were infusion-associated reactions, nasopharyngitis, headache, diarrhea, fatigue, nausea, back pain, pain in extremity, and sinusitis.

Please see full Prescribing Information for Elfabrio.

References: 1. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2024. 2. Fabrazyme. Prescribing Information. Genzyme Corporation; 2024. 3. Germain DP, Linhart A. Pegunigalsidase alfa: a novel, pegylated recombinant alpha-galactosidase enzyme for the treatment of Fabry disease. Front Genet. 2024;15:1395287. doi:10.3389/fgene.2024.1395287. 4. Schiffmann R, Goker-Alpan O, Holida M, et al. Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: a 1-year phase 1/2 clinical trial. J Inherit Metab Dis. 2019;42(3):534-544. doi:10.1002/jimd.12080. 5. Hughes D, Gonzalez D, Maegawa G, et al. Long-term safety and efficacy of pegunigalsidase alfa: a multicenter 6-year study in adult patients with Fabry disease. Genet Med. 2023;25(12):100968. doi:10.1016/j.gim.2023.100968. 6. Wallace EL, Goker-Alpan O, Wilcox WR, et al. Head-to-head trial of pegunigalsidase alfa versus agalsidase beta in patients with Fabry disease and deteriorating renal function: results from the 2-year randomised phase III BALANCE study. J Med Genet. 2024;61(6):520-530. doi:10.1136/jmg-2023-109445. 7. Bernat JA, Hughes D, Linhart A, et al. Assessment of immunogenicity from the pegunigalsidase alfa clinical trial program: integrated analysis of de novo and pre-existing anti-drug antibodies. Poster presented at: WORLDSymposium™ 2024; February 4-9, 2024; San Diego, CA, USA. 8. Mehta A, Wallace EL, Linhart A, et al. Tolerability of pegunigalsidase alfa across the clinical program: integrated analysis of infusion-related reactions by prior enzyme replacement therapy. Poster presented at: WORLDSymposium™ 2024; February 4-9, 2024; San Diego, CA, USA. 9. Hughes D, Bernat JA, Linhart A, et al. Tolerability of pegunigalsidase alfa across the clinical program: integrated analysis of infusion-related reactions by dosing regimens. Poster presented at: WORLDSymposium™ 2024; February 4-9, 2024; San Diego, CA, USA.

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