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Register of orphan medicinal products


GRANTED  

Product information

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

EU orphan designation number: EU/3/18/2026   
Active ingredient: 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
Indication: Treatment of transthyretin-mediated amyloidosis
Sponsor: Alnylam Netherlands B.V.
Strawinskylaan 3051, 1077 ZX, Amsterdam, Nederland

  

European Commission proceduresGoto top of the page

Close date procedure Procedure type EMEA number Decision summary publ decision docs annex
29/05/2018 Orphan designation EMA/OD/019/18 (2018)3394 of 25/05/2018
25/02/2019 Transfer of orphan designation EMA/OD/0000003989 (2019)1637 of 21/02/2019