| Clinical data | |
|---|---|
| Trade names | Pombiliti |
| Other names | ATB-200, ATB200, cipaglucosidase alfa-atga |
| Routes of administration | Intravenous |
| Drug class | Enzyme replacement |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| ChEMBL | |
| Chemical and physical data | |
| Formula | C4489H6817N1197O1298S32 |
| Molar mass | 99347.92 g·mol−1 |
Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease).[3] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.[3]
The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain.[3]
Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.[3]
Medical uses[edit]
Cipaglucosidase alfa is a long-term enzyme replacement therapy used in combination with the enzyme stabilizer miglustat for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency).[3]
Society and culture[edit]
Legal status[edit]
Cipaglucosidase alfa is available in the UK, since June 2021, under the Early Access to Medicines Scheme.[1]
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).[4] The applicant for this medicinal product is Amicus Therapeutics Europe Limited.[4] Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.[3]
Names[edit]
Cipaglucosidase alfa is the international nonproprietary name (INN).[5]
References[edit]
- ^ a b "Cipaglucosidase alfa with miglustat: Treatment protocol: Information for healthcare professionals". Medicines and Healthcare products Regulatory Agency (MHRA). 8 June 2021. Retrieved 18 December 2022.
- ^ "Cipaglucosidase alfa with miglustat in the treatment of late-onset Pompe disease". Medicines and Healthcare products Regulatory Agency (MHRA). 8 June 2021. Retrieved 17 May 2023.
- ^ a b c d e f g "Pombiliti EPAR". European Medicines Agency (EMA). 17 May 2023. Retrieved 17 May 2023. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ a b "Pombiliti: Pending EC decision". European Medicines Agency (EMA). 16 December 2022. Archived from the original on 18 December 2022. Retrieved 18 December 2022. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ^ World Health Organization (2021). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information. 35 (1). hdl:10665/340684.
Further reading[edit]
- Blair HA (May 2023). "Cipaglucosidase Alfa: First Approval". Drugs. 83 (8): 739–745. doi:10.1007/s40265-023-01886-5. PMC 10184071. PMID 37184753.
- 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[edit]
- "Cipaglucosidase alfa with miglustat for treating Pompe disease [ID3771]". NICE. 12 July 2023.
- Clinical trial number NCT03729362 for "PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD" at ClinicalTrials.gov
