Skip to main content
Free research Free research Free compound review with research home and workspace preview
Quick search ChEMBL 36
Compound Detail

CHEMBL3833346

TOFERSEN
💊 Approved Drug
Enter ChEMBL ID:
Free Research Context This compound will appear in recent viewed items on the research home for this browser.
Research home View demo workspace
💊 Approved Drug
No structure available

Basic Information

ChEMBL ID CHEMBL3833346
Name TOFERSEN
Phase Approved
Structure Type SEQ
Therapeutic ✓ Yes

Known Names

BIIB-067 BIIB067 ISIS-SOD1Rx Qalsody Tofersen
0
Targets
0
Activities
0
Assay Types
0
PK Parameters
0
Publications
5
Known Names
1
Indications
1
Mechanisms

Drug Information

Molecule Type Oligonucleotide
First Approval 2023
Availability Prescription
Chirality Single Enantiomer

Routes of Administration

Parenteral

Flags

First in Class
USAN Stem -rsen
USAN Year 2017

Mechanism of Action

Mechanism Action Type Target Direct Efficacy
SOD1 mRNA antisense inhibitor ANTISENSE INHIBITOR SOD1 mRNA

Indications

Amyotrophic Lateral Sclerosis

ATC Classification

N07XX22
tofersen
Level 1: NERVOUS SYSTEM
Level 2: OTHER NERVOUS SYSTEM DRUGS

Data from ChEMBL 36 via Core Engine