MHRA paediatric investigation plan
acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid) (acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid))
MHRA paediatric investigation plan MHRA-100482-PIP01-22 · Agreement
Key facts
| PIP number | MHRA-100482-PIP01-22 |
|---|---|
| Active substances | acetyl-L-leucine ((s)-(acetylamino)-4-methylpentanoic acid) |
| Therapeutic areas | Neurology |
| Conditions | Treatment of Niemann-Pick Disease type C (NPC) |
| Decision | P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) |
| Published | Feb 2, 2023 |
| 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: