Spinal Muscular Atrophy Pipeline 2025: Comprehensive Analysis Of Emerging Therapies, Clinical Trials, Drug Development, And Future Treatment Opportunities

DelveInsight's “Spinal Muscular Atrophy Pipeline Insight 2025” report provides comprehensive insights about 18+ companies and 20+ pipeline drugs in the Spinal Muscular Atrophy pipeline landscape. It covers the Spinal Muscular Atrophy pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Spinal Muscular Atrophy pipeline therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Spinal Muscular Atrophy Pipeline Report

• On September 23, 2025, Scholar Rock (NASDAQ: SRRK), a global biopharmaceutical company committed to improving the lives of children and adults with spinal muscular atrophy (SMA) and other rare, severe neuromuscular diseases through its expertise in myostatin biology and musculoskeletal health, announced that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) for the Biologics License Application (BLA) for apitegromab, intended for the treatment of patients with SMA.
• DelveInsight's Spinal Muscular Atrophy pipeline report depicts a robust space with 18+ active players working to develop 20+ pipeline therapies for Spinal Muscular Atrophy treatment.
• The leading Spinal Muscular Atrophy Companies such as Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.
• Promising Spinal Muscular Atrophy Pipeline Therapies such as OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, vesemnogene lantuparvovec, Olesoxime, NMD670 and others.

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The Spinal Muscular Atrophy Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Spinal Muscular Atrophy Pipeline Report also highlights the unmet needs with respect to the Spinal Muscular Atrophy.

Spinal Muscular Atrophy Overview

Spinal muscular atrophy (SMA) is a genetic disease affecting the central nervous system, peripheral nervous system, and voluntary muscle movement (skeletal muscle). Most of the nerve cells that control muscles are located in the spinal cord, which accounts for the word spinal in the name of the disease. SMA is muscular because its primary effect is on muscles, which don't receive signals from these nerve cells.

Spinal Muscular Atrophy Emerging Drugs Profile

• Apitegromab: Scholar Rock

Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The US Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD) and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) and Orphan Medicinal Product designations, to apitegromab for the treatment of SMA. Currently, it is in phase III stage of development.

• ACTX-401: Alcyone Therapeutics

ACTX-401 is an AAV9-delivered gene replacement therapy for the treatment of a rare form of Spinal Muscular Atrophy (SMA) called SMA with Respiratory Distress type 1 (SMARD1), and for Charcot-Marie-Tooth type 2S (CMT2S). SMARD1 and CMT2S are autosomal recessive genetic disorders caused by mutations in the gene that produces immunoglobulin mu-binding protein 2 (IGHMBP2) and are part of a larger family of disorders called IGHMBP2-related disorders (IRDs). ACTX-401 delivers a functional copy of the IGHMBP2 gene to patients, restoring expression of functional IGHMBP2. ACTX-401 clinical success in SMARD1 and CMT2S could lead to applicability to other IRDs in the future. Currently, it is being investigated in Phase I/II stage of development for the treatment of spinal muscular atrophy.

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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 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.

Spinal Muscular Atrophy Companies

Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.

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 Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy

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Scope of the Spinal Muscular Atrophy Pipeline Report

• Coverage- Global
• Spinal Muscular Atrophy Companies- Ionis Pharmaceuticals, Scholar Rock, Biogen, Astellas Pharma, Alcyone Therapeutics, AndroScience Corporation, Hanugen Theraputics, Voyager Therapeutics, Hoffmann-La Roche, Catalyst Pharmaceuticals, NMD Pharma, Biohaven Pharmaceuticals, CANbridge Pharmaceuticals Inc., Aurimed Pharma, Exicure, Amylon Therapeutics, Amniotics and others.
• Spinal Muscular Atrophy Pipeline Therapies- OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, vesemnogene lantuparvovec, Olesoxime, NMD670 and others.
• Spinal Muscular Atrophy Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• Spinal Muscular Atrophy Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

For a detailed overview of our latest research findings and future plans, read the full details of Spinal Muscular Atrophy Pipeline on our website @ Spinal Muscular Atrophy Emerging Drugs and Companies

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