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 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]
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 is indicated for long-term enzyme replacement therapy in aduls with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). [2] [4]
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]
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 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]
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 is indicated for long-term enzyme replacement therapy in aduls with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase). [2] [4]
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]