Spinal Muscular Atrophy Pipeline 2025: FDA Updates, Therapy Innovations, and Clinical Trial Landscape Analysis by DelveInsight | Genentech Inc, Chugai Pharma, Cytokinetics Inc., Ionis Pharma, Genzyme

(Las Vegas, Nevada, United States) As per DelveInsight's assessment, globally, Spinal Muscular Atrophy pipeline constitutes 18+ key companies continuously working towards developing 20+ Spinal Muscular Atrophy treatment therapies, analysis of Clinical Trials, Therapies, Mechanism of Action, Route of Administration, and Developments analyzes DelveInsight.

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 [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]" 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:

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Companies across the globe are diligently working toward developing novel Spinal Muscular Atrophy treatment therapies with a considerable amount of success over the years.

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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

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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.

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In June 2025, Roche announced that the European Commission (EC) has approved a label extension for Evrysdi (risdiplam), introducing a room-temperature stable 5mg tablet for spinal muscular atrophy (SMA). The 6.5mm tablet can be swallowed whole or dispersed in water, taken with or without food, and stored without refrigeration. Designed for home administration, Evrysdi remains a convenient, non-invasive, disease-modifying therapy for individuals with SMA.

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In June 2025, Biogen Inc. (Nasdaq: BIIB) announced topline data from its Phase 1 study of salanersen (BIIB115/ION306), an investigational antisense oligonucleotide (ASO) for spinal muscular atrophy (SMA). Developed to provide greater potency than SPINRAZA (nusinersen) while working through the same mechanism, salanersen is intended to offer strong efficacy with a single annual dose. Interim results, which also included SMA patients with prior gene therapy, support advancement into registrational studies. Both 40 mg and 80 mg once-yearly doses were well-tolerated and showed significant neuroprotection, evidenced by neurofilament decline and promising functional gains, including attainment of new WHO milestones.

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In March 2025, Novartis' investigational therapy OAV101 IT, an intrathecal version of the approved gene therapy onasemnogene abeparvovec (Zolgensma), has demonstrated encouraging outcomes in improving patient scores on the Hammersmith Functional Motor Scale Expanded (HFMSE). The results, from the Phase 3 STEER trial (NCT05089656), were recently shared at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference.

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In March 2025, Novartis announced favorable efficacy and safety results from its Phase III clinical program evaluating OAV101 IT (onasemnogene abeparvovec), an investigational intrathecal therapy for spinal muscular atrophy (SMA) in patients aged two to under 18 years. In the pivotal Phase III STEER trial, treatment with OAV101 IT led to a 2.39-point increase on the Hammersmith Functional Motor Scale Expanded (HFMSE), compared to a 0.51-point gain in the sham control group-highlighting a significant improvement in motor function specific to SMA.

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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.

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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.

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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.

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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.

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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.

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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.

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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 rare genetic disorder that affects the motor neurons in the spinal cord, leading to progressive muscle weakness and atrophy. It is caused by mutations in the SMN1 gene, which is responsible for producing a protein essential for motor neuron survival. Without enough of this protein, the nerve cells controlling muscles deteriorate, resulting in difficulty with movement, breathing, and swallowing. SMA ranges in severity, with Type 1 being the most severe and appearing in infancy. Early diagnosis and treatment, including gene therapies and SMN-enhancing drugs, can significantly improve quality of life and outcomes.

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Emerging Spinal Muscular Atrophy Drugs Under Different Phases of Clinical Development Include:

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SKG 0201: Skyline Therapeutics

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Spinal muscular atrophy gene therapy: Exegenesis Bio

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BIIB115: Biogen

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GC101: GeneCradle Inc.

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EXG001-307: Hangzhou Jiayin Biotech Ltd

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Apitegromab: Scholar Rock, Inc.

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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

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Oral

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Parenteral

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Intravenous

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Subcutaneous

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Topical

Spinal Muscular Atrophy Molecule Type

Spinal Muscular Atrophy Products have been categorized under various Molecule types, such as

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Monoclonal Antibody

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Peptides

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Polymer

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Small molecule

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Gene therapy

Spinal Muscular Atrophy Pipeline Therapeutics Assessment

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Spinal Muscular Atrophy Assessment by Product Type

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Spinal Muscular Atrophy By Stage and Product Type

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Spinal Muscular Atrophy Assessment by Route of Administration

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Spinal Muscular Atrophy By Stage and Route of Administration

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Spinal Muscular Atrophy Assessment by Molecule Type

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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

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Late-stage products (Phase III)

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Mid-stage products (Phase II)

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Early-stage product (Phase I)

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Pre-clinical and Discovery stage candidates

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Discontinued & Inactive candidates

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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 [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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

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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.

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It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Spinal Muscular Atrophy Treatment.

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Spinal Muscular Atrophy key companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.

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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.

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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.

Download Sample PDF Report to know more about Spinal Muscular Atrophy drugs and therapies [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Spinal Muscular Atrophy Pipeline Market Drivers

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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

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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

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Coverage: Global

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Key Spinal Muscular Atrophy Companies: Skyline Therapeutics, Exegenesis Bio, Biogen, GeneCradle Inc., Hangzhou Jiayin Biotech Ltd, Scholar Rock, Inc., Biohaven Pharmaceuticals, Inc, and others

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Key Spinal Muscular Atrophy Therapies: SKG 0201, Spinal muscular atrophy gene therapy, BIIB115, GC101, EXG001-307, Apitegromab, Talditercept alfa, and others

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Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and Spinal Muscular Atrophy emerging therapies

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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 [https://www.delveinsight.com/sample-request/spinal-muscular-atrophy-sma-pipeline-insight?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

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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