MHRA paediatric investigation plan
Pombiliti (Cipaglucosidase alfa)
MHRA paediatric investigation plan MHRA-100129-PIP01-21-M02 (update) · Modification
Key facts
| PIP number | MHRA-100129-PIP01-21-M02 (update) |
|---|---|
| Product names | Pombiliti, Pombilti |
| Active substances | Cipaglucosidase alfa |
| Therapeutic areas | Endocrinology-Gynaecology-Fertility-metabolism |
| Conditions | Treatment of glycogen storage disease Type II (Pompe's disease) |
| Decision | PM: decision on the application for modification of an agreed paediatric investigation plan. |
| Published | Apr 8, 2025 |
| MHRA record | cms.mhra.gov.uk |
About MHRA paediatric investigation plans
A paediatric investigation plan sets out the studies a company must run in children to support authorisation of a medicine in the UK, or records a waiver or deferral.
Source: UK Medicines and Healthcare products Regulatory Agency (MHRA). Contains public sector information licensed under the Open Government Licence v3.0 · Source date: