Pegunigalsidase alfa
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| Trade names | Elfabrio |
| Other names | PRX-102, pegunigalsidase alfa-iwxj |
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| Routes of administration |
Intravenous |
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| Legal status |
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| Identifiers | |
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| 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. It is a recombinant human α-galactosidase-A. It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.
The most common side effects are infusion-related reactions, hypersensitivity and asthenia.
Pegunigalsidase alfa was approved for medical use in both the European Union and the United States in May 2023.
Medical uses
Pegunigalsidase alfa is indicated for long-term enzyme replacement therapy in aduls with a confirmed diagnosis of Fabry disease (deficiency of alpha-galactosidase).
Society and culture
Legal status
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. The applicant for this medicinal product is Chiesi Farmaceutici S.p.A. Elfabrio was approved for medical use in the European Union in May 2023.
Further reading
- 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.