SMA
1
Pipeline Programs
2
Companies
1
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
Monoclonal Antibody
150%
Gene Therapy
150%
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Scholar RockMA - Cambridge
1 programApitegromabN/AMonoclonal Antibody1 trial
Active Trials
NCT06877689No Longer Available
Clinical Trials (1)
EAP of Apitegromab for Patients With Spinal Muscular Atrophy
N/ANo Longer Available
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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
1 late-stage (Phase 3) programs, potential near-term approvals
Monoclonal Antibody is the dominant modality (50% of programs)
2 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.