Tofersen

Summary

Tofersen (CHEMBL3833346) is an approved oligonucleotide (ATC N07XX22); indicated across 1 condition including amyotrophic lateral sclerosis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: N07XX22
• Indications: 1 condition
• Clinical trials: 8

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3833346
Name	Tofersen
Type	Oligonucleotide
Max phase	4
ATC	N07XX22

Also known as: BIIB-067, BIIB067, ISIS-SOD1Rx, Qalsody, Tofersen, TOFERSEN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

1 indication (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
amyotrophic lateral sclerosis	4	MONDO:0004976	MONDO:0004976

Clinical trials

Total trials: 8.

Phase distribution

Phase	Trials
PHASE3	3
Not specified	2
PHASE4	1
PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07223723	PHASE4	RECRUITING	A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)
NCT04856982	PHASE3	ACTIVE_NOT_RECRUITING	A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation
NCT02623699	PHASE3	COMPLETED	An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)
NCT03070119	PHASE3	COMPLETED	Long-Term Evaluation of BIIB067 (Tofersen)
NCT07294144	PHASE2	RECRUITING	Tofersen in Non-SOD1 ALS
NCT03764488	PHASE1	COMPLETED	A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults
NCT07259980	Not specified	RECRUITING	A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD-1) Amyotrophic Lateral Sclerosis (ALS)
NCT04972487	Not specified	APPROVED_FOR_MARKETING	Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).