Pegunigalsidase alfa

Pegunigalsidase alfa
Names
Trade namesElfabrio
Other namesPRX-102, pegunigalsidase alfa-iwxj
Clinical data
Drug classEnzyme replacement therapy[1]
Main usesFabry disease[2][1]
Side effectsInfusion-related reactions, allergic reactions, weakness[1]
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Routes of
use		Intravenous
Typical dose1 mg/kg q 2 wks[2]
Legal
License data
Legal status
Chemical and physical data
FormulaC2060H3130N552O601S27
Molar mass46110.58 g·mol−1

Pegunigalsidase alfa, sold under the brand name Elfabrio, is a medication used to treat Fabry disease.[2][1] It is given by gradual injection into a vein.[1]

Common side effects include infusion-related reactions, allergic reactions, and weakness.[1] Other side effects may include kidney problems, specifically membranoproliferative glomerulonephritis.[2] It works as enzyme replacement therapy, being a modified form of α-galactosidase-A.[2][1]

Pegunigalsidase alfa was approved for medical use in Europe and the United States in 2023.[1][2] It is made by recombinant DNA technology.[1] As of mid 2023 the cost was unclear in the United States.[4]

Medical uses

Pegunigalsidase alfa is indicated for long-term enzyme replacement therapy in adulds with a confirmed Fabry disease (deficiency of alpha-galactosidase).[2][1]

Dosage

The usual dose is 1 mg/kg every 2 weeks.[2]

Society and culture

On 23 February 2023, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Elfabrio, intended for the treatment of Fabry disease.[1] The applicant for this medicinal product is Chiesi Farmaceutici S.p.A.[5][6] Elfabrio was approved for medical use in the European Union in May 2023.[1]

References

  1. 1 2 3 4 5 6 7 8 9 10 11 12 13 "Elfabrio EPAR". European Medicines Agency. 8 May 2023. Archived from the original on 10 May 2023. Retrieved 9 May 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  2. 1 2 3 4 5 6 7 8 9 "Elfabrio- pegunigalsidase alfa injection, solution, concentrate". DailyMed. 23 May 2023. Archived from the original on 25 May 2023. Retrieved 24 May 2023.
  3. ↑ "Novel Drug Approvals for 2023". U.S. Food and Drug Administration (FDA). 9 May 2023. Archived from the original on 21 January 2023. Retrieved 12 May 2023.
  4. ↑ MSc, Vincent Xie (15 May 2023). "Elfabrio: The New Treatment for Fabry Disease". Xtalks. Archived from the original on 1 July 2023. Retrieved 22 June 2023.
  5. ↑ "Elfabrio: Pending EC decision". European Medicines Agency. 23 February 2023. Archived from the original on 24 February 2023. Retrieved 25 February 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  6. ↑ "Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease" (Press release). Chiesi Global Rare Diseases. 24 February 2023. Archived from the original on 24 February 2023. Retrieved 25 February 2023 – via PR Newswire.
Identifiers:
  • Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, et al. (May 2019). "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". Journal of Inherited Metabolic Disease. 42 (3): 534–544. doi:10.1002/jimd.12080. PMID 30834538. S2CID 73489645.
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