Key facts

Active substance
Synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2’-fluoro and thirty-five 2’-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (ALN-65492)
Therapeutic area
Cardiovascular diseases
Decision number
PIP number
Pharmaceutical form(s)
Solution for injection
Condition(s) / indication(s)
Treatment of transthyretin-related amyloidosis (ATTR amyloidosis)
Route(s) of administration
Subcutaneous use
Contact for public enquiries
Alnylam Netherlands BV

E-mail: clinicaltrials@alnylam.com
Tel. +1 8663 300326

Decision type
W: decision granting a waiver in all age groups for all conditions/indications


How useful was this page?

Add your rating