Pegunigalsidase alfa

Pegunigalsidase alfa
Clinical data
Trade namesElfabrio
Other namesPRX-102, pegunigalsidase alfa-iwxj
AHFS/Drugs.comMonograph
MedlinePlusa623031
License data
Routes of
administration		Intravenous
Drug classLysosomal enzymes
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC2060H3130N552O601S27
Molar mass46110.58 g·mol−1

Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease.[2][3] It is a recombinant human α-galactosidase-A.[3] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.[2]

The most common side effects are infusion-related reactions, hypersensitivity and asthenia.[3]

Pegunigalsidase alfa was approved for medical use in both the European Union and the United States in May 2023.[3][4]

Medical uses

Pegunigalsidase alfa is indicated for long-term enzyme replacement therapy in adults with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).[2][3]

Society and culture

In February 2023, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Elfabrio, intended for the treatment of Fabry disease.[3] 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.[3]

References

  1. ^ "Elfabrio Product information". Health Canada. 9 December 2025. Retrieved 2 January 2026.
  2. ^ a b c d "Elfabrio- pegunigalsidase alfa injection, solution, concentrate". DailyMed. 23 May 2023. Retrieved 24 May 2023.
  3. ^ a b c d e f g h "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.
  4. ^ "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.
  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.

Further reading

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