MHRA paediatric investigation plan
Not available at present (DTX401 (a nonreplicating, recombinant adeno-associated virus serotype 8 vector that contains a codon-optimized, wild-type human glucose-6-phosphatase (G6PC) gene))
MHRA paediatric investigation plan MHRA-100178-PIP01-21 · Agreement
Key facts
| PIP number | MHRA-100178-PIP01-21 |
|---|---|
| Product names | Not available at present |
| Active substances | DTX401 (a nonreplicating, recombinant adeno-associated virus serotype 8 vector that contains a codon-optimized, wild-type human glucose-6-phosphatase (G6PC) gene) |
| Therapeutic areas | Endocrinology-Gynaecology-Fertility-metabolism |
| Conditions | Treatment of glycogen storage disease type 1a (GSDIa) |
| Decision | P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) |
| Published | Sep 27, 2022 |
| 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: