The Spinal Muscular Atrophy Pipeline report embraces in-depth commercial and clinical assessment of the pipeline products from the pre-clinical developmental phase to the marketed phase. The report also covers a detailed description of the drug, including the mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, collaborations, mergers acquisition, funding, designations, and other product-related details.
“Spinal Muscular Atrophy Pipeline Insight, 2025“ report by DelveInsight outlines comprehensive insights into the present clinical development scenario and growth prospects across the Spinal Muscular Atrophy Market.
Some of the key takeaways from the Spinal Muscular Atrophy Pipeline Report:
Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years.
Spinal Muscular Atrophy companies working in the treatment market are Skyline Therapeutics, Exegenesis Bio, Biogen, GeneCradle Inc., Hangzhou Jiayin Biotech Ltd, Scholar Rock, Inc., Biohaven Pharmaceuticals, Inc, and others, are developing therapies for the Spinal Muscular Atrophy treatment
Emerging Spinal Muscular Atrophy therapies in the different phases of clinical trials are- SKG 0201, Spinal muscular atrophy gene therapy, BIIB115, GC101, EXG001-307, Apitegromab, Talditercept alfa, and others are expected to have a significant impact on the Spinal Muscular Atrophy market in the coming years.
In March 2025, Scholar Rock’s spinal muscular atrophy (SMA) treatment demonstrated improvements in muscle function among children under 12 in a key clinical study, yet the results failed to excite investors. The Phase III SAPPHIRE trial (NCT05156320) assessed the safety and effectiveness of apitegromab, a monoclonal antibody designed to enhance muscle growth by targeting and inhibiting the inactive forms of myostatin specifically within skeletal muscle.
In February 2025, A pilot clinical trial suggested that spinal cord stimulation (SCS) may help restore function in patients with spinal muscular atrophy (SMA) by gradually activating dormant motor neurons and enhancing leg muscle strength. Conducted by the University of Pittsburgh School of Medicine (NCT05430113), the study evaluated Medtronic’s Vectris SureScan device in three SMA patients. All participants demonstrated improved mobility, each increasing their distance in the six-minute walk test (6MWT)—a key indicator of muscle endurance—by at least 20 meters after three months of treatment.
In January 2025, Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on developing novel therapies for spinal muscular atrophy (SMA), cardiometabolic conditions, and other serious diseases involving protein growth factors, has announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. This muscle-directed therapy is intended to deliver significant motor function improvement in individuals with SMA who are already receiving SMN-targeted treatments. The company also plans to submit a Marketing Authorisation Application to the European Medicines Agency in the first quarter of 2025.
In January 2025, Analytics firm GlobalData has expressed a positive outlook on the future of Novartis’ gene therapy for spinal muscular atrophy (SMA), onasemnogene abeparvovec (OAV101 IT). The therapy may soon be accessible to a wider patient group, thanks to encouraging results from Novartis’ Phase III STEER trial (NCT05089656), in which the intrathecal version of the treatment successfully met its primary endpoint.
In November 2024, Biohaven Ltd. (NYSE: BHVN), a global clinical-stage biopharmaceutical company dedicated to developing and commercializing transformative therapies for various rare and common diseases, announced updates on the development programs for taldefgrobep alfa in Spinal Muscular Atrophy (SMA) and obesity.
In November 2024, Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company dedicated to developing innovative therapies for spinal muscular atrophy (SMA), cardiometabolic conditions, and other severe diseases driven by protein growth factors, has announced positive and statistically significant topline results from the pivotal Phase 3 SAPPHIRE trial. These findings highlight the potential of apitegromab to transform the standard of care for SMA patients.
In April 2024, Exegenesis Bio, a fast-expanding global leader in genetic medicine, has shared clinical efficacy and safety findings from its Phase 1/2 trial (EXG001-307) for Spinal Muscular Atrophy (SMA) Type I. The data was presented on May 8, 2024, during the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting in Baltimore, Maryland (poster #627).
Spinal Muscular Atrophy Overview
Spinal Muscular Atrophy (SMA) is a genetic neuromuscular disorder characterized by the progressive degeneration of motor neurons in the spinal cord and brainstem. Motor neurons are nerve cells responsible for controlling voluntary muscle movement. When these motor neurons degenerate, muscles weaken and atrophy, leading to difficulties with movement, muscle weakness, and potentially life-threatening respiratory problems.
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Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include:
SKG 0201: Skyline Therapeutics
Spinal muscular atrophy gene therapy: Exegenesis Bio
BIIB115: Biogen
GC101: GeneCradle Inc.
EXG001-307: Hangzhou Jiayin Biotech Ltd
Apitegromab: Scholar Rock, Inc.
Talditercept alfa: Biohaven Pharmaceuticals, Inc
Spinal Muscular Atrophy Route of Administration
Spinal Muscular Atrophy pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs, such as
Oral
Parenteral
Intravenous
Subcutaneous
Topical
Spinal Muscular Atrophy Molecule Type
Spinal Muscular Atrophy Products have been categorized under various Molecule types, such as
Monoclonal Antibody
Peptides
Polymer
Small molecule
Gene therapy
Spinal Muscular Atrophy Pipeline Therapeutics Assessment
Spinal Muscular Atrophy Assessment by Product Type
Spinal Muscular Atrophy By Stage and Product Type
Spinal Muscular Atrophy Assessment by Route of Administration
Spinal Muscular Atrophy By Stage and Route of Administration
Spinal Muscular Atrophy Assessment by Molecule Type
Spinal Muscular Atrophy by Stage and Molecule Type
DelveInsight’s Spinal Muscular Atrophy Report covers around 20+ products under different phases of clinical development like
Late-stage products (Phase III)
Mid-stage products (Phase II)
Early-stage product (Phase I)
Pre-clinical and Discovery stage candidates
Discontinued & Inactive candidates
Route of Administration
Further Spinal Muscular Atrophy product details are provided in the report. Download the Spinal Muscular Atrophy pipeline report to learn more about the emerging Spinal Muscular Atrophy therapies
Some of the key companies in the Spinal Muscular Atrophy Therapeutics Market include:
Key companies developing therapies for Spinal Muscular Atrophy are – Genentech Inc, Chugai Pharmaceutical, Cytokinetics Inc., Ionis Pharmaceuticals Inc., Genzyme Corporation, Novartis International AG, Astellas Pharma Inc., Takeda Pharmaceutical Company Limited, PerkinElmer Inc., Pfizer Inc., UW Health, Novo Nordisk A/S, Cure SMA, Boehringer Ingelheim Gmbh, Biogen Inc, F. Hoffmann-La Roche AG, Catalyst Pharmaceutical, Salarius Pharmaceuticals Inc, Regeneron Pharmaceuticals Inc, Isis Pharmaceuticals Inc., PTC Therapeutics, Natera Inc, AstraZeneca PLC, and others.
Spinal Muscular Atrophy Pipeline Analysis:
The Spinal Muscular Atrophy pipeline report provides insights into
The report provides detailed insights about companies that are developing therapies for the treatment of Spinal Muscular Atrophy with aggregate therapies developed by each company for the same.
It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment.
Spinal Muscular Atrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
Spinal Muscular Atrophy Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Spinal Muscular Atrophy market.
The report is built using data and information traced from the researcher’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations, and featured press releases from company/university websites and industry-specific third-party sources, etc.
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Spinal Muscular Atrophy Pipeline Market Drivers
Increase in prevalence of Spinal Muscular Atrophy, increasing number of clinical trials and government funding for R&D are some of the important factors that are fueling the Spinal Muscular Atrophy Market.
Spinal Muscular Atrophy Pipeline Market Barriers
However, lack of public knowledge of this rare medical condition, cost associated with the treatment and other factors are creating obstacles in the Spinal Muscular Atrophy Market growth.
Scope of Spinal Muscular Atrophy Pipeline Drug Insight
Coverage: Global
Key Spinal Muscular Atrophy Companies: Skyline Therapeutics, Exegenesis Bio, Biogen, GeneCradle Inc., Hangzhou Jiayin Biotech Ltd, Scholar Rock, Inc., Biohaven Pharmaceuticals, Inc, and others
Key Spinal Muscular Atrophy Therapies: SKG 0201, Spinal muscular atrophy gene therapy, BIIB115, GC101, EXG001-307, Apitegromab, Talditercept alfa, and others
Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies
Spinal Muscular Atrophy Market Dynamics: Spinal Muscular Atrophy market drivers and Spinal Muscular Atrophy market barriers
Request for Sample PDF Report for Spinal Muscular Atrophy Pipeline Assessment and clinical trials
Table of Contents
1. Spinal Muscular Atrophy Report Introduction
2. Spinal Muscular Atrophy Executive Summary
3. Spinal Muscular Atrophy Overview
4. Spinal Muscular Atrophy- Analytical Perspective In-depth Commercial Assessment
5. Spinal Muscular Atrophy Pipeline Therapeutics
6. Spinal Muscular Atrophy Late Stage Products (Phase II/III)
7. Spinal Muscular Atrophy Mid Stage Products (Phase II)
8. Spinal Muscular Atrophy Early Stage Products (Phase I)
9. Spinal Muscular Atrophy Preclinical Stage Products
10. Spinal Muscular Atrophy Therapeutics Assessment
11. Spinal Muscular Atrophy Inactive Products
12. Company-University Collaborations (Licensing/Partnering) Analysis
13. Spinal Muscular Atrophy Key Companies
14. Spinal Muscular Atrophy Key Products
15. Spinal Muscular Atrophy Unmet Needs
16 . Spinal Muscular Atrophy Market Drivers and Barriers
17. Spinal Muscular Atrophy Future Perspectives and Conclusion
18. Spinal Muscular Atrophy Analyst Views
19. Appendix
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.
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