Cipaglucosidase alfa
| Clinical data | |
|---|---|
| Other names | ATB-200, ATB200 |
| Routes of administration |
Intravenous |
| Drug class | Enzyme replacement |
| ATC code | |
| Legal status | |
| Legal status |
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| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| ChEMBL | |
| Chemical and physical data | |
| Formula | C4489H6817N1197O1298S32 |
| Molar mass | 99347.92 g·mol−1 |
Cipaglucosidase alfa is an experimental enzyme replacement therapy medication for the treatment of glycogen storage disease type II (Pompe disease). Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.
Society and culture
Legal status
Cipaglucosidase alfa is available in the UK, since June 2021, under the Early Access to Medicines Scheme.
On 15 December 2022, 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 Pombiliti, intended for the treatment of glycogen storage disease type II (Pompe disease). The applicant for this medicinal product is Amicus Therapeutics Europe Limited.
Names
Cipaglucosidase alfa is the international nonproprietary name (INN).
Further reading
- Schoser B, Roberts M, Byrne BJ, Sitaraman S, Jiang H, Laforêt P, et al. (December 2021). "Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial". The Lancet. Neurology. 20 (12): 1027–1037. doi:10.1016/S1474-4422(21)00331-8. PMID 34800400. S2CID 244304730.
External links
- "Cipaglucosidase alfa with miglustat for treating Pompe disease [ID3771]". NICE.
- Clinical trial number NCT03729362 for "PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD" at ClinicalTrials.gov