MHRA paediatric investigation plan
Fitusiran (Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues)
MHRA paediatric investigation plan MHRA-101283-PIP01-23-M02 (update) · Modification
Key facts
| PIP number | MHRA-101283-PIP01-23-M02 (update) |
|---|---|
| Product names | Fitusiran |
| Active substances | Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues |
| Therapeutic areas | Haematology-Hemostaseology |
| Conditions | Treatment of haemophilia A, Treatment of Haemophilia B |
| Decision | PM: decision on the application for modification of an agreed paediatric investigation plan. |
| Published | Mar 10, 2025 |
| 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: