Pegunigalsidase alfa

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]

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: ℞-only[1][2] EU: Rx-only[3]
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.^[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 adults 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]