MHRA paediatric investigation plan
Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203) (Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203))
MHRA paediatric investigation plan MHRA-100330-PIP01-21 · Agreement
Key facts
| PIP number | MHRA-100330-PIP01-21 |
|---|---|
| Active substances | Autologous CD34+ hematopoietic stem and progenitor cells (HSPCs) genetically modified with the lentiviral vector IDUA LVV, encoding for the human α-L-iduronidase (IDUA) gene (OTL-203) |
| Therapeutic areas | Other: Metabolic disorders |
| Conditions | Treatment of Mucopolysaccharidosis type I, Hurler syndrome (MPS-IH) |
| Decision | P: decision agreeing on a paediatric investigation plan, with or without partial waiver(s) or deferrals(s) |
| Published | Jul 29, 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: