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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.

Indication

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

Please see Full Prescribing Information for Elfabrio.

References: 1. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2023. 2. 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. 3. US Food and Drug Administration. Drug approval package: Elfabrio; Multi-discipline Review. May 8, 2023. Accessed July 18, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/761161Orig1s000MultidisciplineR.pdf. 4. Wallace EL, Goker-Alpan O, Wilcox WR, et al. First results of a head-to-head trial of pegunigalsidase alfa vs agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study. Poster presented at: WORLDSymposium: 19th Annual Research Meeting; February 22-26, 2023; Orlando, FL. https://protalixbiotherapeutics.gcs-web.com/static-files/58f1efe6-de3c-455d-b814-3e9e96373a8c. 5. Atta M, Hughes D, Gonzalez D, et al. Long-term safety and efficacy of pegunigalsidase alfa: a multicenter extension study in adult patients with Fabry disease. Poster presented at: 7th Update on Fabry Disease: Biomarkers, Progression and Treatment Opportunities in 2022; May 29-31, 2022; Würzburg, Germany. https://protalixbiotherapeutics.gcs-web.com/static-files/465d2891-4135-42e5-8581-f24e175cb07f.

References: 1. Rombach SM, Smid BE, Bouwman MG, et al. Long term enzyme replacement therapy for Fabry disease: effectiveness on kidney, heart and brain. Orphanet J Rare Dis. 2013;8:47. doi:10.1186/1750-1172-8-47. 2. Weidemann F, Neimann M, Stork S, et al. Long-term outcome of enzyme-replacement therapy in advanced Fabry disease: evidence for disease progression towards serious complications. J Intern Med. 2013;274(4):331-341. doi:10.1111/joim.12077. 3. Desnick RJ, Brady R, Barranger J, et al. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med. 2003;138(4):338-346. doi:10.7326/0003-4819-138-4-200302180-00014. 4. Waldek S, Feriozzi S. Fabry nephropathy: a review – how can we optimize the management of Fabry nephropathy? BMC Nephrol. 2014;15:72. doi:10.1186/1471-2369-15-72. 5. Hoffman B, Mayatepek E. Fabry disease—often seen, rarely diagnosed. Dtsch Arztebl Int. 2009;106(26):440-447. doi:10.3238/arztebl.2009.0440. 6. Mehta A, Hughes DA. Fabry Disease. In: Adam MP, Everman DB, Mirzaa GM, et al, eds. GeneReviews®. Seattle (WA): University of Washington, Seattle; August 5, 2002. 7. Ortiz A, Germain DP, Desnick RJ, et al. Fabry disease revisited: management and treatment recommendations for adult patients. Mol Genet Metab. 2018;123(4):416-427. doi:10.1016/j.ymgme.2018.02.014. 8. Schiffmann R, Warnock DG, Banikazemi M, et al. Fabry disease: progression of nephropathy, and prevalence of cardiac and cerebrovascular events before enzyme replacement therapy. Nephrol Dial Transplant. 2009;24(7):2102-2111. doi:10.1093/ndt/gfp031. 9. Wilcox WR, Oliveira JP, Hopkin RJ, et al. Females with Fabry disease frequently have major organ involvement: lessons from the Fabry Registry. Mol Genet Metab. 2008;93(2):112-128. doi:10.1016/j.ymgme.2007.09.013. 10. Wang RY, Lelis A, Mirocha J, Wilcox WR. Heterozygous Fabry women are not just carriers, but have a significant burden of disease and impaired quality of life. Genet Med. 2007;9(1):34-45. doi:10.1097/gim.0b013e31802d8321. 11. Morand O, Johnson J, Walter J, et al. Symptoms and quality of life in patients with Fabry disease: results from an international patient survey. Adv Ther. 2019;36(10):2866-2880. doi:10.1007/s12325-019-01061-x. 12. Germain DP. Fabry disease. Orphanet J Rare Dis. 2010;5:30. doi:10.1186/1750-1172-5-30.

References: 1. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2023. 2. 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. 3. Wallace EL, Goker-Alpan O, Wilcox WR, et al. First results of a head-to-head trial of pegunigalsidase alfa vs agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study. Poster presented at: WORLDSymposium: 19th Annual Research Meeting; February 22-26, 2023; Orlando, FL. https://protalixbiotherapeutics.gcs-web.com/static-files/58f1efe6-de3c-455d-b814-3e9e96373a8c. 4. Atta M, Hughes D, Gonzalez D, et al. Long-term safety and efficacy of pegunigalsidase alfa: a multicenter extension study in adult patients with Fabry disease. Poster presented at: 7th Update on Fabry Disease: Biomarkers, Progression and Treatment Opportunities in 2022; May 29-31, 2022; Würzburg, Germany. https://protalixbiotherapeutics.gcs-web.com/static-files/465d2891-4135-42e5-8581-f24e175cb07f. 5. US Food and Drug Administration. Drug approval package: Elfabrio; Multi-discipline Review. May 8, 2023. Accessed July 18, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/761161Orig1s000MultidisciplineR.pdf. 6. Bernat J, Holida M, Longo N, et al. Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: 2-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study. Poster presented at: WORLDSymposium: 19th Annual Research Meeting; February 22-26, 2023; Orlando, FL. https://protalixbiotherapeutics.gcs-web.com/static-files/d2dae305-1926-4418-9c62-3da527a62395. 7. West M, Linhart A, Dostalova G, et al. Switching from agalsidase alfa to pegunigalsidase alfa to treat patients with Fabry disease: 1 year of treatment data from BRIDGE, a phase 3 open-label study. Poster presented at: 7th Update on Fabry Disease: Biomarkers, Progression and Treatment Opportunities in 2022; May 29-31, 2022; Würzburg, Germany. https://protalixbiotherapeutics.gcs-web.com/static-files/e7e1cbc9-4858-42a2-b03f-1854e3e7a143.

References: 1. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2023. 2. Wallace EL, Goker-Alpan O, Wilcox WR, et al. First results of a head-to-head trial of pegunigalsidase alfa vs agalsidase beta in Fabry disease: 2-year results of the phase 3 randomized, double-blind, BALANCE study. Poster presented at: WORLDSymposium: 19th Annual Research Meeting; February 22-26, 2023; Orlando, FL. https://protalixbiotherapeutics.gcs-web.com/static-files/58f1efe6-de3c-455db814-3e9e96373a8c. 3. US Food and Drug Administration. Drug approval package: Elfabrio; Multi-discipline Review. May 8, 2023. Accessed July 18, 2023. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2023/761161Orig1s000MultidisciplineR.pdf.

References: 1. Atta M, Hughes D, Gonzalez D, et al. Long-term safety and efficacy of pegunigalsidase alfa: a multicenter extension study in adult patients with Fabry disease. Poster presented at: 7th Update on Fabry Disease: Biomarkers, Progression and Treatment Opportunities in 2022; May 29-31, 2022; Würzburg, Germany. https://protalixbiotherapeutics.gcs-web.com/static-files/465d2891-4135-42e5-8581-f24e175cb07f. 2. Elfabrio. Prescribing Information. Chiesi Farmaceutici S.p.A.; 2023. 3. Data on file. Chiesi Farmaceutici S.p.A.; December 2012. 4. Data on file. Chiesi Farmaceutici S.p.A.; January 2016. 5. 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.

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