Spinal Muscular Atrophy - Pipeline Insight, 2025

This “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 pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

• Global coverage

Spinal muscular atrophy Understanding

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. Atrophy is the medical term for getting smaller, which is what generally happens to muscles when they’re not stimulated by nerve cells. SMA involves the loss of nerve cells called motor neurons in the spinal cord and is classified as a motor neuron disease. In the most common form of SMA (chromosome 5 SMA, or SMN-related SMA), there is wide variability in age of onset, symptoms, and rate of progression. In order to account for these differences, chromosome 5-related SMA, which often is autosomal recessive, is classified into types 1 through 4. The age at which SMA symptoms begin roughly correlates with the degree to which motor function is affected: The earlier the age of onset, the greater the impact on motor function. Children who display symptoms at birth or in infancy typically have the lowest level of functioning (type 1). Later-onset SMA with a less severe course (types 2 and 3, and in teens or adults, type 4) generally correlates with increasingly higher levels of motor function.

'Spinal muscular atrophy - Pipeline Insight, 2025' report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Spinal muscular atrophy pipeline landscape is provided which includes the disease overview and Spinal muscular atrophy treatment guidelines. The assessment part of the report embraces, in depth Spinal muscular atrophy commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Spinal muscular atrophy collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Spinal muscular atrophy R&D. The therapies under development are focused on novel approaches to treat/improve Spinal muscular atrophy.

Spinal muscular atrophy Emerging Drugs Chapters

This segment of the Spinal muscular atrophy report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Spinal muscular atrophy Emerging Drugs

• 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.

Spinal muscular atrophy: Therapeutic Assessment

This segment of the report provides insights about the different Spinal muscular atrophy drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Spinal muscular atrophy

There are approx. 18+ key companies which are developing the therapies for Spinal muscular atrophy. The companies which have their Spinal muscular atrophy drug candidates in the most advanced stage, i.e. phase III include, Scholar Rock.

Phases

The 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) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates

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.

Molecule Type

Products have been categorized under various Molecule types such as

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

Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Spinal muscular atrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Spinal muscular atrophy therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Spinal muscular atrophy drugs.

Spinal muscular atrophy Report Insights

• Spinal muscular atrophy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Spinal muscular atrophy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Spinal muscular atrophy drugs?
• How many Spinal muscular atrophy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Spinal muscular atrophy?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Spinal muscular atrophy therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Spinal muscular atrophy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• 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
• Exegenesis Bio

Key Products

• Apitegromab
• BIIB 110
• Reldesemtiv
• ACTX 401
• ASCJ 9
• Reldesemtiv
• RO7204239
• NMD-670
• CNS specific amniotic mesenchymal stem cell therapy

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