MHRA paediatric investigation plan
Aqneursa (acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid))
MHRA paediatric investigation plan MHRA-100482-PIP01-22-M02 (update) · Modification
Key facts
| PIP number | MHRA-100482-PIP01-22-M02 (update) |
|---|---|
| Product names | Aqneursa |
| Active substances | acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid) |
| Therapeutic areas | Neurology |
| Conditions | Treatment of Niemann-Pick Disease Type C (NPC) |
| Decision | PM: decision on the application for modification of an agreed paediatric investigation plan. |
| Published | Oct 28, 2024 |
| 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: