Spinal Muscular Atrophy

Rare Diseases
20
Pipeline Programs
13
Companies
35
Clinical Trials
6 recruiting
2
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
4
2
5
0
6
3
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
10100%
+ 20 programs with unclassified modality

On Market (2)

Approved therapies currently available

Roche
EVRYSDIApproved
risdiplam
Roche
Survival of Motor Neuron 2 Splicing Modifier [EPC]oral2020
Biogen
SPINRAZAApproved
nusinersen
Biogen
Survival Motor Neuron-2-directed RNA Interaction [EPC]intrathecal2016

Competitive Landscape

11 companies ranked by most advanced pipeline stage

Biogen
BiogenCAMBRIDGE, MA
15 programs
2
1
2
4
1
NusinersenPhase 3RNA Therapeutic1 trial
NusinersenPhase 3RNA Therapeutic5 trials
SalanersenPhase 3RNA Therapeutic1 trial
nusinersenPhase 3RNA Therapeutic1 trial
NusinersenPhase 2RNA Therapeutic1 trial
+10 more programs
Active Trials
NCT05109637Completed93Est. Jul 2023
NCT03300869Unknown300Est. Dec 2019
NCT04292574Recruiting800Est. May 2025
+16 more trials
Genentech
GenentechCA - Oceanside
2 programs
1
RisdiplamPhase 41 trial
Spinal Cord StimulatorN/A1 trial
Active Trials
NCT06300996Active Not Recruiting3Est. Sep 2029
NCT05522361Active Not Recruiting10Est. Jun 2026
Roche
RocheSTAVANGER NORWAY, Norway
2 programs
1
1
RisdiplamPhase 21 trial
test for SMN1 exon 7 deletionN/A
Active Trials
NCT03032172Completed174Est. Feb 2025
Biohaven
BiohavenNEW HAVEN, CT
2 programs
1
taldefgrobep alfaPhase 31 trial
Optimized rehabilitation programN/A1 trial
Active Trials
NCT06419322Active Not Recruiting14Est. Dec 2026
NCT05337553Active Not RecruitingEst. Jun 2026
Novartis
NovartisBASEL, Switzerland
2 programs
1
1
OAV101Phase 35 trials
branaplamPhase 1/22 trials
Active Trials
NCT05330286Terminated9Est. Aug 2022
NCT02268552Completed40Est. Dec 2022
NCT07448610Not Yet Recruiting36Est. Apr 2034
+4 more trials
Leadiant Biosciences
Leadiant BiosciencesMD - Rockville
2 programs
1
1
Valproic Acid and LevocarnitinePhase 2
Valproic AcidPhase 1
NMD Pharma
NMD PharmaDenmark - Aarhus
1 program
1
NMD670Phase 21 trial
Active Trials
NCT05794139Recruiting54Est. May 2026
Abbott
AbbottABBOTT PARK, IL
1 program
1
Valproic AcidPhase 11 trial
Active Trials
NCT00374075Completed42Est. Feb 2006
M&
Merck & Co.RAHWAY, NJ
1 program
Confirmatory TestingN/A
Genomics
GenomicsUK - Oxford
1 program
Prospective Evaluation of Infants With Spinal Muscular Atrophy:N/A1 trial
Active Trials
NCT02831296Unknown1,000
Alcyone Therapeutics
1 program
ThecaFlex DRx SystemN/A

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
NovartisOAV101
GenentechRisdiplam
NovartisOAV101
BiogenSalanersen
NovartisOAV101
Biohaventaldefgrobep alfa
NovartisOAV101
BiogenNusinersen
NovartisOAV101
BiogenNusinersen
BiogenNusinersen
Biogennusinersen
NMD PharmaNMD670
RocheRisdiplam
BiogenNusinersen

Showing 15 of 35 trials with date data

Clinical Trials (35)

Total enrollment: 140,294 patients across 35 trials

NCT07448610NovartisOAV101

ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)

Start: Jul 2026Est. completion: Apr 203436 patients
Phase 4Not Yet Recruiting
NCT05522361GenentechRisdiplam

Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen

Start: Nov 2022Est. completion: Jun 202610 patients
Phase 4Active Not Recruiting
NCT05073133NovartisOAV101

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
NCT07444476BiogenSalanersen

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam

Start: Apr 2026Est. completion: Jun 203290 patients
Phase 3Not Yet Recruiting
NCT05335876NovartisOAV101

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 in Clinical Trials

Start: Dec 2022Est. completion: Feb 2031175 patients
Phase 3Recruiting
NCT05337553Biohaventaldefgrobep alfa

A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy

Start: Jul 2022Est. completion: Jun 2026
Phase 3Active Not Recruiting
NCT05089656NovartisOAV101

Efficacy and Safety of Intrathecal OAV101 (AVXS-101) in Pediatric Patients With Type 2 Spinal Muscular Atrophy (SMA)

Start: Feb 2022Est. completion: Apr 2025126 patients
Phase 3Completed
NCT05067790BiogenNusinersen

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)

Start: Jan 2022Est. completion: Jun 202745 patients
Phase 3Active Not Recruiting
NCT04851873NovartisOAV101

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

Start: Sep 2021Est. completion: Jun 202324 patients
Phase 3Completed
NCT02594124BiogenNusinersen

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

Start: Nov 2015Est. completion: Aug 2023292 patients
Phase 3Completed
NCT02292537BiogenNusinersen

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

Start: Nov 2014Est. completion: Feb 2017126 patients
Phase 3Completed
NCT02193074Biogennusinersen

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

Start: Aug 2014Est. completion: Nov 2016122 patients
Phase 3Terminated
NCT05794139NMD PharmaNMD670

Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy

Start: Sep 2023Est. completion: May 202654 patients
Phase 2Recruiting
NCT03032172RocheRisdiplam

A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy

Start: Mar 2017Est. completion: Feb 2025174 patients
Phase 2Completed
NCT02462759BiogenNusinersen

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

Start: Aug 2015Est. completion: Sep 201821 patients
Phase 2Terminated
NCT02386553BiogenNusinersen

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

Start: May 2015Est. completion: Dec 202425 patients
Phase 2Completed
NCT01839656Biogennusinersen

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

Start: May 2013Est. completion: Aug 201721 patients
Phase 2Completed
NCT02268552Novartisbranaplam

An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA)

Start: Apr 2015Est. completion: Dec 202240 patients
Phase 1/2Completed
NCT01703988BiogenNusinersen

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

Start: Oct 2012Est. completion: Jan 201534 patients
Phase 1/2Completed
NCT05330286Novartisbranaplam

Study to Compare the Pharmacokinetics, Safety and Tolerability of the Pediatric and Adult Branaplam Formulation in Healthy Adults and the Effect of Food on the Latter.

Start: Apr 2022Est. completion: Aug 20229 patients
Phase 1Terminated
NCT02052791Biogennusinersen

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)

Start: Jan 2014Est. completion: Jan 201747 patients
Phase 1Completed
NCT01780246Biogennusinersen

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)

Start: Jan 2013Est. completion: Feb 201418 patients
Phase 1Completed
NCT01494701BiogenNusinersen

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

Start: Nov 2011Est. completion: Jan 201328 patients
Phase 1Completed
NCT00374075AbbottValproic Acid

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

Start: Sep 2003Est. completion: Feb 200642 patients
Phase 1Completed
NCT06955897BiogenNusinersen

Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA

Start: Apr 2025Est. completion: Feb 202745 patients
N/ARecruiting
NCT06419322BiohavenOptimized rehabilitation program

Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients With Spinal Muscular Atrophy (SMA).

Start: Jun 2024Est. completion: Dec 202614 patients
N/AActive Not Recruiting
NCT06300996GenentechSpinal Cord Stimulator

Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb

Start: May 2024Est. completion: Sep 20293 patients
N/AActive Not Recruiting
NCT05789758BiogenNusinersen

A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies

Start: Dec 2023Est. completion: Oct 203320 patients
N/ARecruiting
NCT05866419BiogenThecaFlex DRx System

Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy

Start: Nov 2023Est. completion: Mar 202790 patients
N/ARecruiting
NCT05109637BiogenKonectom NMD Application

A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders

Start: Feb 2022Est. completion: Jul 202393 patients
N/ACompleted
NCT04591678Biogennusinersen

Adults With SMA Treated With Nusinersen

Start: Oct 2018Est. completion: Jun 202115 patients
N/ACompleted
NCT03554343Biogentest for SMN1 exon 7 deletion

Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium

Start: Mar 2018Est. completion: Feb 2021136,339 patients
N/ACompleted
NCT03300869BiogenNatural History of Types 2 and 3 SMA in Taiwan

Natural History of Types 2 and 3 SMA in Taiwan

Start: Sep 2017Est. completion: Dec 2019300 patients
N/AUnknown
NCT02831296GenomicsProspective Evaluation of Infants With Spinal Muscular Atrophy:

Prospective Evaluation of Infants With Spinal Muscular Atrophy:

Start: Feb 20161,000 patients
N/AUnknown
NCT04292574BiogenPatient Registry

UK SMA Patient Registry

Start: Jul 2008Est. completion: May 2025800 patients
N/ARecruiting

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

6 late-stage (Phase 3) programs — potential near-term approvals
6 actively recruiting trials targeting 140,294 patients
13 companies competing in this space

Related Indications

Cystic Fibrosis(244)Hemophilia A(139)Sickle Cell Disease(64)Fabry Disease(55)Duchenne Muscular Dystrophy(53)Hemophilia B(47)Phenylketonuria(32)Gaucher Disease(29)Severe Hemophilia A(26)Pompe Disease(20)