Chemistry:Cipaglucosidase alfa
| 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 | |
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| 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
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
Legal status
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
Cipaglucosidase alfa is the international nonproprietary name (INN).[5]
References
- ↑ 1.0 1.1 "Cipaglucosidase alfa with miglustat: Treatment protocol: Information for healthcare professionals". 8 June 2021. https://www.gov.uk/government/publications/cipaglucosidase-alfa-with-miglustat-in-the-treatment-of-late-onset-pompe-disease/cipaglucosidase-alfa-with-miglustat-treatment-protocol-information-for-healthcare-professionals.
- ↑ "Cipaglucosidase alfa with miglustat in the treatment of late-onset Pompe disease". 8 June 2021. https://www.gov.uk/government/publications/cipaglucosidase-alfa-with-miglustat-in-the-treatment-of-late-onset-pompe-disease.
- ↑ 3.0 3.1 3.2 3.3 3.4 3.5 3.6 "Pombiliti EPAR". 17 May 2023. https://www.ema.europa.eu/en/medicines/human/EPAR/pombiliti. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ 4.0 4.1 "Pombiliti: Pending EC decision". 16 December 2022. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/pombiliti. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 85". WHO Drug Information 35 (1). 2021.
Further reading
- "Cipaglucosidase Alfa: First Approval". Drugs 83 (8): 739–745. May 2023. doi:10.1007/s40265-023-01886-5. PMID 37184753.
- "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. December 2021. doi:10.1016/S1474-4422(21)00331-8. PMID 34800400.
External links
- "Cipaglucosidase alfa with miglustat for treating Pompe disease [ID3771"]. 12 July 2023. https://www.nice.org.uk/guidance/indevelopment/gid-ta10887.
- Clinical trial number NCT03729362 for "PROPEL Study - A Study Comparing ATB200/AT2221 With Alglucosidase/Placebo in Adult Subjects With LOPD" at ClinicalTrials.gov
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