Other alimentary tract and metabolism products (A16)
| 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]
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]
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]
Cipaglucosidase alfa is the international nonproprietary name (INN).[5]
