Biology:Pegunigalsidase alfa
| 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 | |
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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.[1][3] It is a recombinant human α-galactosidase-A.[3] It is a hydrolytic lysosomal neutral glycosphingolipid-specific enzyme.[1]
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][2]
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).[1][3]
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.[3] The applicant for this medicinal product is Chiesi Farmaceutici S.p.A.[4][5] Elfabrio was approved for medical use in the European Union in May 2023.[3]
References
- ↑ 1.0 1.1 1.2 1.3 "Elfabrio- pegunigalsidase alfa injection, solution, concentrate". 23 May 2023. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=6fb674bf-744e-431f-92ef-b2a5a66c8cf3.
- ↑ 2.0 2.1 "Novel Drug Approvals for 2023". 9 May 2023. https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023.
- ↑ 3.0 3.1 3.2 3.3 3.4 3.5 3.6 3.7 "Elfabrio EPAR". 8 May 2023. https://www.ema.europa.eu/en/medicines/human/EPAR/elfabrio. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "Elfabrio: Pending EC decision". 23 February 2023. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/elfabrio. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "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
- "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. May 2019. doi:10.1002/jimd.12080. PMID 30834538.
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