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Muscular Atrophy, Spinal

3
Pipeline Programs
9
Companies
15
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
1
0
0
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
8100%
+ 16 programs with unclassified modality

Competitive Landscape

8 companies ranked by most advanced pipeline stage

Sandoz
SandozAustria - Kundl
3 programs
1
OAV101Phase 41 trial
A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular N/A1 trial
Newborn Screening for Spinal Muscular AtrophyN/A1 trial
Active Trials
NCT07403214Completed4,805Est. Jun 2025
NCT05481164Completed33,568Est. Jul 2025
NCT05073133Completed16Est. Aug 2023
Genentech
GenentechCA - Oceanside
2 programs
1
OlesoximePhase 21 trial
Newborn Screening for Spinal Muscular AtrophyN/A
Active Trials
NCT02628743Completed131Est. Dec 2018
Alcyone Therapeutics
1 program
1
NusinersenPhase 1RNA Therapeutic
Biogen
BiogenCAMBRIDGE, MA
9 programs
Adult Spinal Muscular Atrophy (SMA) China RegistryN/A1 trial
Nusinersen Sodium InjectionN/ARNA Therapeutic1 trial
Nusinersen Sodium InjectionN/ARNA Therapeutic1 trial
Pediatric Spinal Muscular Atrophy (SMA) China RegistryN/A1 trial
NusinersenPHASE_1RNA Therapeutic1 trial
+4 more programs
Active Trials
NCT05618379Active Not Recruiting200Est. Jun 2028
NCT04317794Active Not Recruiting74Est. Feb 2027
NCT04419233Completed50Est. Nov 2023
+6 more trials
Roche
RocheSTAVANGER NORWAY, Norway
6 programs
RisdiplamPHASE_1
OlesoximePHASE_2
RisdiplamPHASE_2
RisdiplamPHASE_2
RisdiplamPHASE_4
+1 more programs
Novartis
NovartisBASEL, Switzerland
1 program
Newborn Screening for Spinal Muscular AtrophyN/A
Catalyst Pharmaceuticals
Catalyst PharmaceuticalsCORAL GABLES, FL
1 program
Amifampridine PhosphatePHASE_21 trial
Active Trials
NCT03781479Completed13Est. Jul 2020
Novo Nordisk
Novo NordiskBAGSVAERD DENMARK, Denmark
1 program
somatotropinPHASE_21 trial
Active Trials
NCT00533221Completed20Est. Aug 2011

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
SandozOAV101
BiogenNusinersen
BiogenSalanersen
BiogenNusinersen
BiogenNusinersen
Catalyst PharmaceuticalsAmifampridine Phosphate
GenentechOlesoxime
Novo Nordisksomatotropin
BiogenNusinersen
SandozA Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular
BiogenAdult Spinal Muscular Atrophy (SMA) China Registry
SandozNewborn Screening for Spinal Muscular Atrophy
BiogenPediatric Spinal Muscular Atrophy (SMA) China Registry
BiogenNusinersen Sodium Injection
BiogenNusinersen Sodium Injection

Clinical Trials (15)

Total enrollment: 39,883 patients across 15 trials

Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA)

Start: Nov 2021Est. completion: Aug 202316 patients
Phase 4Completed

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)

Start: Jan 2021Est. completion: Oct 202546 patients
Phase 4Completed

A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec

Start: Sep 2026Est. completion: Jul 203342 patients
Phase 3Not Yet 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)

Start: Apr 2021Est. completion: Jul 2026115 patients
Phase 3Active Not Recruiting

Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy

Start: Mar 2020Est. completion: May 2024145 patients
Phase 3Completed

Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients

Start: Jan 2019Est. completion: Jul 202013 patients
Phase 2Completed

A Study to Evaluate Long Term Safety, Tolerability, and Effectiveness of Olesoxime in Patients With Spinal Muscular Atrophy (SMA)

Start: Jan 2016Est. completion: Dec 2018131 patients
Phase 2Completed

Pilot Study of Growth Hormon to Treat SMA Typ II and III

Start: Oct 2007Est. completion: Aug 201120 patients
Phase 2Completed

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)

Start: Jan 2025Est. completion: Jun 202758 patients
Phase 1Recruiting
NCT07403214SandozA Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular

A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy

Start: Jun 2025Est. completion: Jun 20254,805 patients
N/ACompleted
NCT05618379BiogenAdult Spinal Muscular Atrophy (SMA) China Registry

Adult Spinal Muscular Atrophy (SMA) China Registry

Start: Jan 2023Est. completion: Jun 2028200 patients
N/AActive Not Recruiting
NCT05481164SandozNewborn Screening for Spinal Muscular Atrophy

Newborn Screening for Spinal Muscular Atrophy

Start: Mar 2022Est. completion: Jul 202533,568 patients
N/ACompleted
NCT05042921BiogenPediatric Spinal Muscular Atrophy (SMA) China Registry

Pediatric Spinal Muscular Atrophy (SMA) China Registry

Start: Nov 2021Est. completion: Sep 2027600 patients
N/AActive Not Recruiting
NCT04419233BiogenNusinersen Sodium Injection

Non-Interventional, Postmarketing Surveillance Study of Nusinersen Sodium Injection

Start: Nov 2020Est. completion: Nov 202350 patients
N/ACompleted
NCT04317794BiogenNusinersen Sodium Injection

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

Start: Jul 2019Est. completion: Feb 202774 patients
N/AActive Not Recruiting

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

1 actively recruiting trials targeting 39,883 patients
9 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.