| Clinical data | |
|---|---|
| Trade names | Elfabrio |
| Other names | PRX-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 | |
| Formula | C2060H3130N552O601S27 |
| Molar mass | 46110.58 g·mol−1 |
Pegunigalsidase alfa, sold under the brand nameElfabrio, is anenzyme replacement therapy for the treatment ofFabry disease.[2][4] It is a recombinant humanα-galactosidase-A.[4] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.[2]
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]
Pegunigalsidase alfa isindicated for long-term enzyme replacement therapy in adults with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).[2][4]
On 23 February 2023, theCommittee for Medicinal Products for Human Use (CHMP) of theEuropean 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]
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