MHRA paediatric investigation plan
Not available at present (messenger RNA encoding Cas9, single guide RNA targeting the human TTR gene)
MHRA paediatric investigation plan MHRA-100848-PIP01-23 · Waiver
Key facts
| PIP number | MHRA-100848-PIP01-23 |
|---|---|
| Product names | Not available at present |
| Active substances | messenger RNA encoding Cas9, single guide RNA targeting the human TTR gene |
| Therapeutic areas | Neurology, Cardiovascular Diseases |
| Conditions | Treatment for transthyretin amyloidosis (ATTR) |
| Decision | W: decision granting a waiver in all age groups for the listed condition(s). |
| Published | Dec 19, 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: