Pegunigalsidase alfa

Enzyme replacement therapy medication From Wikipedia, the free encyclopedia

Pegunigalsidase alfa, sold under the brand name Elfabrio, is an enzyme replacement therapy for the treatment of Fabry disease.[2][4] It is a recombinant human α-galactosidase-A.[4] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.[2]

Quick Facts Clinical data, Trade names ...
Pegunigalsidase alfa
Clinical data
Trade namesElfabrio
Other namesPRX-102, pegunigalsidase alfa-iwxj
License data
Routes of
administration
Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC2060H3130N552O601S27
Molar mass46110.58 g·mol−1
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The most common side effects are infusion-related reactions, hypersensitivity and asthenia.[4]

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

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][4]

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.[4] 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.[4]

References

Further reading

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