Key Facts

PIPS Key Facts
Active Substance
Active Substance:
  • eplontersen
Invented Name
Not available at present
PIP Number MHRA-100801-PIP01-22
Pharmaceutical form(s)
Pharmaceutical form(s):
  • Solution for injection
Therapeutic area
Therapeutic area:
  • Neurology
Conditions / Indications
Conditions / Indications:
  • Treatment of transthyretin-mediated amyloidosis
Route(s) of administration
Route(s) of administration:
  • Subcutaneous 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
Compliance Check Decision Date
Compliance Check Procedure Number

Decision Document

PIPS Decision Documents
Decision Document Decision Document on a Paediatric Investigation Plan (PIP):eplontersen.pdf
Published Date 24/08/2023