Pegunigalsidase alfa

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]

Pegunigalsidase alfa

Clinical data
Trade names	Elfabrio
Other names	PRX-102, pegunigalsidase alfa-iwxj
License data	US DailyMed: Pegunigalsidase alfa
Routes of administration	Intravenous
ATC code	A16AB20 (WHO)
Legal status
Legal status	US: WARNING[1]Rx-only[2][3] EU: Rx-only[4]
Identifiers
CAS Number	1644392-61-9
DrugBank	DB14992
UNII	8M7V7Q6537
KEGG	D11685
Chemical and physical data
Formula	C2060H3130N552O601S27
Molar mass	46110.58 g·mol−1

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

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.^[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

1. "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
2. "Elfabrio- pegunigalsidase alfa injection, solution, concentrate". DailyMed. 23 May 2023. Retrieved 24 May 2023.
3. "Novel Drug Approvals for 2023". U.S. Food and Drug Administration (FDA). 9 May 2023. Archived from the original on 21 January 2023. Retrieved 12 May 2023.
4. "Elfabrio EPAR". European Medicines Agency. 8 May 2023. Archived from the original on 10 May 2023. Retrieved 9 May 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
5. "Elfabrio: Pending EC decision". European Medicines Agency. 23 February 2023. Archived from the original on 24 February 2023. Retrieved 25 February 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
6. "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

• 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.