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Spinraza

(Nusinersen)

ApprovedRNA therapy

Nusinersen is used to treat spinal muscular atrophy (SMA), a rare genetic disease that weakens muscles and affects movement. It works by helping the body produce more of a protein needed for nerve and muscle function.[2] Spinraza is currently approved and in use across 4 indications[4].[1]

Spinraza is one of 31 aso programs targeting Spinal Muscular Atrophy, Type IV. It is currently in Approved.

4

Indications

28

Total Trials

7

Active Trials

30

Competitors

1

FDA Application

NDA/BLA/ANDA regulatory filings

7

Patents

Pipeline

Status by Indication[4][1]

Indication

Spinal Muscular Atrophy, Type IVActive, not recruiting

ALSApproved

Spinal Muscular AtrophyActive, not recruiting

Proximal Spinal Muscular AtrophyApproved

Spinal Muscular Atrophy, Type IVPreclinical

Active, not recruiting

ALSApproved

Approved

Spinal Muscular AtrophyPhase 3

Active, not recruiting

Proximal Spinal Muscular AtrophyApproved

Approved

Competition

Competitive Landscape

35 competitors in Spinal Muscular Atrophy

#DrugCompanyPhaseTrials

Evrysdi

Spinraza

Prilosec

DDexcel Pharma Technologies Ltd.

Amondys 45

Sarepta Therapeutics

Depakote

Droxia

Rilutek

AAzurity Pharmaceuticals

Celebrex

PPhiladelphia Eye Associates

Onasemnogene Abeparvovec

RO7204239

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q3 2026

Phase 3Active, not recruiting

Spinal Muscular Atrophy

NCT04729907

Q4 2026

Phase 3Active, not recruiting

Spinal Muscular Atrophy

NCT04089566

Q1 2027

Active, not recruiting

Study completion

NCT04317794

Q2 2027

Adult

NCT06978985

Phase 3Active, not recruiting

Spinal Muscular Atrophy

NCT05067790

Phase 1Recruiting

Spinal Muscular Atrophy

NCT06555419

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy20 trials

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)

Active, not recruiting· Biogen logo

Biogen·Phase 3·NCT05067790·45 enrolled

Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments

Withdrawn·NNYU Langone Health·Phase 1·NCT04050852

A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA)

Completed· Biogen logo

Biogen·Phase 2·NCT01839656·21 enrolled

Show 17 more trials

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Recruiting·SScholar Rock, Inc.·Phase 2·NCT07047144·52 enrolled

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)

Active, not recruiting· Biogen logo

Biogen·Phase 3·NCT04729907·115 enrolled

Antisense Oligonucleotide for Spinal Muscular Atrophy

Unknown status·FFirst Affiliated Hospital of Fujian Medical University··NCT05187260·1,000 enrolled

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)

Completed· Biogen logo

Biogen·Phase 1·NCT01780246·18 enrolled

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA)

Completed· Biogen logo

Biogen·Phase 3·NCT02292537·126 enrolled

A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Completed· Biogen logo

Biogen·Phase 2·NCT02386553·25 enrolled

Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

No longer available· Biogen logo

Biogen··NCT02865109

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)

Completed· Biogen logo

Biogen·Phase 4·NCT04488133·46 enrolled

Effect of Nusinersen on Adults With Spinal Muscular Atrophy

Completed·NNorthwell Health··NCT03878030·12 enrolled

An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy

Completed· Biogen logo

Biogen·Phase 1/2·NCT01703988·34 enrolled

An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA)

Completed· Biogen logo

Biogen·Phase 1·NCT01494701·28 enrolled

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)

Completed· Biogen logo

Biogen·Phase 1·NCT02052791·47 enrolled

A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy

Terminated· Biogen logo

Biogen·Phase 3·NCT02193074·122 enrolled

A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA).

Terminated· Biogen logo

Biogen·Phase 2·NCT02462759·21 enrolled

A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies

Completed· Biogen logo

Biogen·Phase 3·NCT02594124·292 enrolled

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)

Recruiting· Biogen logo

Biogen·Phase 1·NCT06555419·58 enrolled

DEVOTE: Nusinersen Higher-Dose in SMA

Active, not recruiting· Biogen logo

Biogen·Phase 3·NCT04089566·145 enrolled

Adult2 trials

Adult SMA Research and Clinical Hub

Recruiting·NNewcastle-upon-Tyne Hospitals NHS Trust··NCT06978985·600 enrolled

Adults With SMA Treated With Nusinersen

Completed·OOhio State University··NCT04591678·15 enrolled

Function1 trial

Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms)

Completed·CCHU de Reims·N/A·NCT04576494·4 enrolled

Children1 trial

Metabolomics of Children With SMA

Completed·UUniversity Medical Centre Ljubljana··NCT04587492·35 enrolled

Treatment1 trial

Hyperkinetic Movements in Patients With Disease of Motor Neurons and Their Response to Treatment With Nusinersen

Unknown status·UUniversity Medical Centre Ljubljana··NCT04825119·110 enrolled

2 more indications

Observational Study1 trial

Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study

Completed·HHospital Israelita Albert Einstein··NCT04404764·155 enrolled

Unspecified2 trials

Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)

Active, not recruiting· Biogen logo

Biogen··NCT04317794·74 enrolled

Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection

Completed· Biogen logo

Biogen··NCT04419233·50 enrolled

Regulatory

FDA Approval Timeline[5]

Dec 23, 2016

SPINRAZA

NDA NDA209531

Sources

References

[1]
ClinicalTrials.gov — 28 trial records for Nusinersen. U.S. National Library of Medicine, National Institutes of Health.
[2]
MedlinePlus Drug Information — Spinraza drug information. U.S. National Library of Medicine, National Institutes of Health.
[3]
ChEMBL — Nusinersen compound record. European Bioinformatics Institute (EMBL-EBI).
[4]
Open Targets Platform — Drug–disease associations for Nusinersen. EMBL-EBI & Wellcome Sanger Institute.
[5]
OpenFDA — FDA approval records (NDA NDA209531). U.S. Food & Drug Administration.
[6]
FDA Orange Book — Patent and exclusivity data for Spinraza. U.S. Food & Drug Administration.
[7]
PubMed — 4 publications indexed for Nusinersen. U.S. National Library of Medicine.
[8]
SEC EDGAR — Biogen company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Mechanism of Action: SMN2 Antisense Oligonucleotide[3]
Modality: RNA therapy[3]
Highest Phase: Approved[1]
Company: Biogen

Patent Landscape[6]

Patent Cliff

Jul 11, 2027

Earliest patent expiration — generic entry possible after this date

Patent #Expires

7838657Jul 11, 2027

9717750Jun 17, 2030

8980853Nov 24, 2030

8361977Dec 23, 2030

9926559Jan 9, 2034

2 more patents

10436802Sep 11, 2035

12013403Mar 4, 2036

Key Publications[7]

Surgical Options for Cranial Nerve Functional Diseases.

Adv Tech Stand NeurosurgJan 1, 2026DOI: 10.1007/978-3-032-22008-0_14

Indications and Limits of Endoscopic Endonasal Approaches in Pediatric Neurosurgery.

Adv Tech Stand NeurosurgJan 1, 2026DOI: 10.1007/978-3-032-22008-0_11

Higher-Dose Nusinersen in Spinal Muscular Atrophy: DEVOTE Phase 3 Interim

N Engl J MedFeb 15, 2023DOI: 10.1056/NEJMoa2209965

Nusinersen in Later-Onset Spinal Muscular Atrophy: Long-Term Results From SHINE

Lancet NeurolApr 20, 2022DOI: 10.1016/S1474-4422(22)00098-1

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