Overview
Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA. Other types of enzyme replacement therapy for the treatment of Pompe disease include alglucosidase alfa and avalglucosidase alfa. Cipaglucosidase alfa is conjugated with mannose-6-phosphate (M6P) N-glycans that bind to the cation-independent mannose-6-phosphate receptor (CI-MPR) in skeletal muscle, one of the main affected tissues in Pompe disease. Compared to alglucosidase alfa, cipaglucosidase alfa has a higher M6P content. In December 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended cipaglucosidase alfa be granted marketing authorization for the treatment of Pompe disease, and the EMA fully approved the drug on March 27, 2023. Cipaglucosidase alfa is coadministered with miglustat, a small-molecule pharmacological chaperone that stabilizes the conformation of the enzyme.
Indication
In Europe, 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, also known as acid α-glucosidase (GAA) deficiency.
Associated Conditions
- Late-onset Pompe Disease
Clinical Trials
Title | Posted | Study ID | Phase | Status | Sponsor |
|---|---|---|---|---|---|
2023/11/07 | N/A | Recruiting | |||
2021/03/22 | Phase 3 | Recruiting | |||
2019/10/24 | Phase 3 | Completed | |||
2019/04/11 | Phase 3 | Recruiting | |||
2016/02/05 | Phase 1 | Completed |
FDA Drug Approvals
Approved Product | Manufacturer | NDC Code | Route | Strength | Effective Date |
|---|---|---|---|---|---|
| AMICUS THERAPEUTICS US, LLC | 71904-200 | INTRAVENOUS | 105 mg in 1 1 | 9/1/2023 |
EMA Drug Approvals
Approved Product | Authorization Holder | Status | Issued Date |
|---|---|---|---|
Authorised | 3/20/2023 |
HSA Drug Approvals
Approved Product | Manufacturer | Approval Number | Dosage Form | Strength | Approval Date |
|---|---|---|---|---|---|
| No HSA approvals found for this drug. | |||||
NMPA Drug Approvals
Approved Product | Company | Approval Number | Drug Type | Dosage Form | Approval Date |
|---|---|---|---|---|---|
| No NMPA approvals found for this drug. | |||||
PPB Drug Approvals
Approved Product | Registration No. | Company | Licence No. | Strength | Registration Date |
|---|---|---|---|---|---|
| No PPB approvals found for this drug. | |||||
TGA Drug Approvals
Approved Product | ARTG ID | Sponsor | Registration Type | Status | Registration Date |
|---|---|---|---|---|---|
| POMBILITI cipaglucosidase alfa 105mg powder for injection vial | 430450 | Medicine | A | 2/17/2025 |
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