| Assessment Status | Rapid Review Complete |
| HTA ID | 25027 |
| Drug | Cipaglucosidase alfa in combination with miglustat |
| Brand | Pombiliti® in combination with Opfolda® |
| Indication | Cipaglucosidase alfa in combination with miglustat is indicated for the treatment of adults with late-onset Pompe disease (acid α-glucosidase [GAA] deficiency). |
| Assessment Process | |
| Rapid review commissioned | 31/03/2025 |
| Rapid review completed | 02/05/2025 |
| Rapid review outcome | A full HTA is not recommended. The NCPE recommends that cipaglucosidase alfa in combination with miglustat not be considered for reimbursement at the submitted price*. |
*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
Next steps: The NCPE Assessment Report and recommendation, will be considered by the HSE when making their decision on reimbursement, while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
Further information on this process may be found here.
Further information on the status of this decision may be found here.
