Pegunigalsidase alfa explained
| Tradename: | Elfabrio |
| Dailymedid: | Pegunigalsidase alfa |
| Routes Of Administration: | Intravenous |
| Atc Prefix: | A16 |
| Atc Suffix: | AB20 |
| Legal Us: | Rx-only |
| Legal Us Comment: | [1] [2] |
| Legal Eu: | Rx-only |
| Cas Number: | 1644392-61-9 |
| Drugbank: | DB14992 |
| Unii: | 8M7V7Q6537 |
| Kegg: | D11685 |
| Synonyms: | PRX-102, pegunigalsidase alfa-iwxj |
| C: | 2060 |
| H: | 3130 |
| N: | 552 |
| O: | 601 |
| S: | 27 |
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).[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. 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.
Further reading
- Schiffmann R, Goker-Alpan O, Holida M, Giraldo P, Barisoni L, Colvin RB, Jennette CJ, Maegawa G, Boyadjiev SA, Gonzalez D, Nicholls K, Tuffaha A, Atta MG, Rup B, Charney MR, Paz A, Szlaifer M, Alon S, Brill-Almon E, Chertkoff R, Hughes D . 6 . 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 . 30834538 . 10.1002/jimd.12080 . 73489645 . free .
Notes and References
- Web site: Elfabrio- pegunigalsidase alfa injection, solution, concentrate . DailyMed . 23 May 2023 . 24 May 2023.
- Web site: Novel Drug Approvals for 2023 . U.S. Food and Drug Administration (FDA) . 9 May 2023 . 12 May 2023 . 21 January 2023 . https://web.archive.org/web/20230121035617/https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2023 . live .
- Web site: Elfabrio EPAR . European Medicines Agency . 8 May 2023 . 9 May 2023 . 10 May 2023 . https://web.archive.org/web/20230510153253/https://www.ema.europa.eu/en/medicines/human/EPAR/elfabrio . live . Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- Web site: Elfabrio: Pending EC decision . European Medicines Agency . 23 February 2023 . 25 February 2023 . 24 February 2023 . https://web.archive.org/web/20230224165603/https://www.ema.europa.eu/en/medicines/human/summaries-opinion/elfabrio . live . 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 . Chiesi Global Rare Diseases . PR Newswire . 24 February 2023 . 25 February 2023 . 24 February 2023 . https://web.archive.org/web/20230224163754/https://www.prnewswire.com/news-releases/chiesi-global-rare-diseases-and-protalix-biotherapeutics-receive-positive-chmp-opinion-for-pegunigalsidase-alfa-for-treatment-of-fabry-disease-301755440.html . live .
