Nusinersen

Summary

Nusinersen (CHEMBL3301572) is an approved oligonucleotide (ATC M09AX07); indicated across 2 conditions including spinal muscular atrophy.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Oligonucleotide
• ATC class: M09AX07
• Indications: 2 conditions
• Clinical trials: 26

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL3301572
Name	Nusinersen
Type	Oligonucleotide
Max phase	4
ATC	M09AX07

Also known as: BIIB-058, BIIB058, ISIS 396443, ISIS-396443, Nusinersen, NUSINERSEN

Parent form; salt/anhydrous children: CHEMBL3833342

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

2 indications (0 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
spinal muscular atrophy	3	MONDO:0001516	EFO:0008525

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 26.

Phase distribution

Phase	Trials
Not specified	9
PHASE3	6
PHASE2	4
PHASE1	4
PHASE4	1
PHASE1/PHASE2	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04488133	PHASE4	COMPLETED	A Study to Learn About the Effect of Nusinersen (BIIB058) Given as Injections to Children With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec (RESPOND)
NCT04729907	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD)
NCT05067790	PHASE3	ACTIVE_NOT_RECRUITING	A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
NCT02193074	PHASE3	TERMINATED	A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy
NCT02292537	PHASE3	COMPLETED	A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)
NCT02594124	PHASE3	COMPLETED	A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies
NCT04089566	PHASE3	COMPLETED	Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy
NCT07047144	PHASE2	RECRUITING	A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
NCT01703988	PHASE1/PHASE2	COMPLETED	An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy
NCT01839656	PHASE2	COMPLETED	A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)
NCT02386553	PHASE2	COMPLETED	A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
NCT02462759	PHASE2	TERMINATED	A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).
NCT06555419	PHASE1	RECRUITING	A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
NCT01494701	PHASE1	COMPLETED	An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)
NCT01780246	PHASE1	COMPLETED	An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701)
NCT02052791	PHASE1	COMPLETED	An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246)
NCT04050852	EARLY_PHASE1	WITHDRAWN	Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments
NCT04317794	Not specified	ACTIVE_NOT_RECRUITING	Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
NCT02865109	Not specified	NO_LONGER_AVAILABLE	Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
NCT03878030	Not specified	COMPLETED	Effect of Nusinersen on Adults With Spinal Muscular Atrophy
NCT04419233	Not specified	COMPLETED	Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection
NCT04576494	Not specified	COMPLETED	Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)
NCT04587492	Not specified	COMPLETED	Metabolomics of Children With SMA
NCT04591678	Not specified	COMPLETED	Adults With SMA Treated With Nusinersen
NCT04825119	Not specified	UNKNOWN	Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen
NCT05187260	Not specified	UNKNOWN	Antisense Oligonucleotide for Spinal Muscular Atrophy

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).