MHRA-100848-PIP01-23

Key Facts

Active Substance
Active Substance:
  • messenger RNA encoding Cas9, single guide RNA targeting the human TTR gene
Invented Name
Not available at present
PIP Number MHRA-100848-PIP01-23
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Suspension for infusion
Therapeutic area
  • Neurology
  • Cardiovascular Diseases
Conditions / Indications
Conditions / Indications:
  • Treatment for transthyretin amyloidosis (ATTR)
Route(s) of administration
Route(s) of administration:
  • Intravenous use
PIP applicant
Decision Type
Decision Type
W: decision granting a waiver in all age groups for the listed condition(s).
Compliance Check
Compliance Check
No
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

Decision Document Decision Document on a Paediatric Investigation Plan (PIP):messenger RNA encoding Cas9, single guide RNA targeting the human TTR gene.pdf
Published Date 19/12/2023