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).[4][5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.[5]
| Clinical data | |
|---|---|
| Trade names | Pombiliti |
| Other names | ATB-200, ATB200, cipaglucosidase alfa-atga |
| AHFS/Drugs.com | Monograph |
| MedlinePlus | a623057 |
| License data | |
| Pregnancy category |
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| 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 |
The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain.[5] The most common side effects of cipaglucosidase alfa in combination with miglustat are headache, diarrhea, fatigue, nausea, abdominal pain, and fever.[6]
Cipaglucosidase alfa was approved for medical use in the European Union in March 2023,[5] and in the United States in September 2023.[6]
Medical uses
editCipaglucosidase 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).[4][5]
Adverse effects
editCipaglucosidase alfa in combination with miglustat may cause serious side effects including life-threatening allergic reactions during and after the infusion and harm to an unborn baby if taken while pregnant.[6]
The most common side effects of cipaglucosidase alfa in combination with miglustat are headache, diarrhea, fatigue, nausea, abdominal pain, and fever.[6]
History
editThe FDA approved cipaglucosidase alfa in combination with miglustat based on evidence from a clinical trial (Trial 1/NCT03729362) of 123 participants with late-onset Pompe disease. Safety data from the use of cipaglucosidase alfa in combination with miglustat was primarily obtained from one clinical trial (Trial 1, NCT03729362).[6] Data from two other trials (Trial 2/NCT02675465 and Trial 3/NCT04138277) were also reviewed for completeness of the safety assessment.[6] The three trials enrolled 151 participants with late-onset Pompe disease.[6] The trials were conducted at 61 sites in 24 countries around the world, including the United States.[6] In Trial 1, 123 adults with late-onset Pompe disease received either cipaglucosidase alfa intravenously once every two weeks for 52 weeks in combination with miglustat, or another medication (called the active comparator) intravenously once every two weeks for 52 weeks in combination with placebo.[6] Of the 123 participants, 95 previously received enzyme replacement therapy, and 28 never received enzyme replacement therapy before the trial.[6] Neither the participants nor the healthcare providers knew which treatment was being given until after Week 52.[6]
Society and culture
editLegal status
editCipaglucosidase alfa is available in the UK, since June 2021, under the Early Access to Medicines Scheme.[2]
In 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).[7] The applicant for this medicinal product is Amicus Therapeutics Europe Limited.[7] Cipaglucosidase alfa was approved for medical use in the European Union in March 2023.[5]
Names
editCipaglucosidase alfa is the international nonproprietary name (INN).[8]
References
edit- ^ "Cipaglucosidase alfa (Pombiliti) Use During Pregnancy". Drugs.com. 8 February 2024. Archived from the original on 18 May 2024. Retrieved 18 May 2024.
- ^ a b "Cipaglucosidase alfa with miglustat: Treatment protocol: Information for healthcare professionals". Medicines and Healthcare products Regulatory Agency (MHRA). 8 June 2021. Archived from the original on 18 December 2022. 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. Archived from the original on 17 May 2023. Retrieved 17 May 2023.
- ^ a b c "Pombiliti - cipaglucosidase alfa-atga injection, powder, lyophilized, for solution". DailyMed. 6 October 2023. Archived from the original on 18 May 2024. Retrieved 18 May 2024.
- ^ 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 c d e f g h i j k "Drug Trials Snapshots: Pombiliti". U.S. Food and Drug Administration. 28 September 2023. Archived from the original on 5 April 2024. Retrieved 18 May 2024.
This article incorporates text from this source, which is in the public domain.
- ^ 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
- Clinical trial number NCT02675465 for "First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221" at ClinicalTrials.gov
- Clinical trial number NCT04138277 for "A Study to Assess the Long-term Safety and Efficacy of ATB200/AT2221 in Adult Subjects With LOPD" at ClinicalTrials.gov
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