Spinal Muscular Atrophy Treatment Market Size in the 7MM is expected to grow a significant CAGR by 2034, estimates DelveInsight | Novartis Pharmaceuticals, Hoffmann-La Roche, Biogen, Catalyst Pharmaceuticals Inc., Lantu Biopharma

DelveInsight's "Spinal Muscular Atrophy Market Insights, Epidemiology and Market Forecast - 2034" report delivers an in-depth understanding of Spinal Muscular Atrophy, historical and forecasted epidemiology as well as the Spinal Muscular Atrophy market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

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Key Takeaways from the Spinal Muscular Atrophy Market Report
• In March 2025, Hoffmann-La Roche conducted a study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.
• In March 2025, Genentech Inc. announced a study to investigate the long-term safety and effectiveness of risdiplam, prescribed based on clinician judgment as per the Evrysdi® U.S. Package Insert (USPI) in adult and pediatric participants with SMA. In this study, participants will be followed for the duration of the study or until withdrawal of consent, loss to follow-up, or death. Participants who discontinue risdiplam may still remain in the study, if they agree to continue participating in the follow-up assessments.
• In March 2025, NMD Pharma A/S organized a study is to evaluate the efficacy, safety, tolerability and pharmacokinetics of NMD670 in the treatment of ambulatory adults with spinal muscular atrophy type 3.
• In January 2025, Scholar Rock announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for apitegromab. This muscle-targeted therapy aims to provide significant motor function improvements in patients with SMA who are undergoing SMN-targeted treatments.
• In January 2025, Biogen announced that the FDA has accepted its supplemental New Drug Application (sNDA) and the EMA has validated the application for a higher dose regimen of nusinersen for spinal muscular atrophy (SMA).
• According to the findings, Spinal Muscular Atrophy has a carrier frequency of 1 in 40-50 and an estimated incidence of 1 in 10,000 live births, Spinal muscular atrophy is the second most common autosomal recessive disorder.
• The traditional types of Spinal Muscular Atrophy are caused by biallelic mutations in the survival motor neuron 1 (SMN1) gene located on chromosome 5 that result in a lack of SMN proteins. A second gene, called SMN2, also produces SMN proteins but they are usually truncated and only 10%-20% are viable.
• It is estimated that roughly 60% of Spinal muscular atrophy newborns have Spinal muscular atrophy type I while types II and III make up the remaining 40%.
• The leading Spinal Muscular Atrophy Companies such as Novartis Pharmaceuticals, Hoffmann-La Roche, Biogen, Catalyst Pharmaceuticals Inc., Lantu Biopharma and others.
• Promising Spinal Muscular Atrophy Pipeline Therapies such as OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, Vesemnogene Lantuparvovec, and others.

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Spinal Muscular Atrophy Epidemiology Segmentation in the 7MM
• Spinal Muscular Atrophy Diagnosed prevalent cases
• Spinal Muscular Atrophy Gender-specific diagnosed prevalent cases
• Spinal Muscular Atrophy Type-specific diagnosed prevalent cases
• Spinal Muscular Atrophy Age-specific diagnosed prevalent cases

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Spinal Muscular Atrophy Marketed Drugs
• SPINRAZA: Biogen
SPINRAZA, manufactured by Biogen is an approved drug for the treatment of Spinal muscular atrophy in adults and children. The active ingredient of the drug is Nusinersen. It is a modified oligonucleotide with 2'¬ hydroxyl groups of the ribofuranosyl rings that are replaced with 2'-O-2-methoxyethyl groups and the phosphate linkages that are replaced with phosphorothioate linkages. Nusinersen targets the intronic splicing silencer N1 (ISS-N1) of exon 7 of the SMN2 transcript. It blocks the ISS-N1 which results in the destabilization of an inhibitory stem-loop RNA structure in intron 7, close to the end of exon 7 and thus leads to increased exon inclusion. The drug is approved across the 7MM for treatment of Spinal muscular atrophy.

• ZOLGENSMA (onasemnogene abeparvovac-xioi): Novartis
ZOLGENSMA is an adeno-associated virus (AAV) vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy with bi-allelic mutations in the survival motor neuron 1 gene. Intravenous administration of ZOLGENSMA results in cell transduction and expression of the survival motor neuron protein was observed in human case studies. This therapy was approved in the US in May 2019 and in Europe and Japan in May 2020 and March 2020, respectively.

• EVRYSDI (risdiplam): Genentech/ F. Hoffmann-La Roche
EVRYSDI is a survival of motor neuron 2 splicing modifier designed to treat patients with spinal muscular atrophy caused by mutations in chromosome 5q that lead to survival of motor neuron protein deficiency in patients two months of age and older. The drug received US FDA approval in August 2020 and the EC granted marketing authorization to the drug in March 2021.

Spinal Muscular Atrophy Emerging Drugs
• Apitegromab (SRK-015): Scholar Rock
Apitegromab (SRK-015) is a selective inhibitor of the activation of latent myostatin and is an investigational product candidate for treating patients with Spinal muscular atrophy. Rather than the traditional approach of blocking already-activated, mature myostatin or the receptor, Apitegromab selectively targets the precursor, or inactive, form of myostatin to block its activation in the muscle. The US FDA has granted Orphan Drug Designation, Fast Track designation, and Rare Pediatric Disease designation while the European Commission also granted Orphan Medicinal Product Designation to the drug and the EMA has granted Priority Medicines (PRIME) Designation to apitegromab for the treatment of Spinal muscular atrophy.

• Taldefgrobep Alfa (BHV-2000): Biohaven/ Bristol-Myers Squibb
Taldefgrobep Alfa, a myostatin inhibitor, myostatin is a natural protein that normally works to regulate skeletal muscle growth, an important process in healthy muscular development. In patients with neuromuscular diseases, over-active myostatin can critically limit the growth needed to achieve normal developmental and functional milestones. Myostatin inhibition is a potential therapeutic strategy for children and adults with a range of neuromuscular conditions for whom active myostatin can limit the skeletal muscle growth needed to achieve developmental and functional milestones.

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Spinal Muscular Atrophy Market Dynamics
The spinal muscular atrophy market dynamics are anticipated to change in the coming years. Presently, there exist three FDA-approved treatments for spinal muscular atrophy: Spinraza, Evrysdi, and Zolgensma. Among these options, Zolgensma stands out as a life-saving gene therapy that has gained approval in various international markets known as the 7MM.

Scope of the Spinal Muscular Atrophy Market Report
• Coverage- 7MM
• Study Period- 2020-2034
• Spinal Muscular Atrophy Companies- Novartis Pharmaceuticals, Hoffmann-La Roche, Biogen, Catalyst Pharmaceuticals Inc., Lantu Biopharma and others.
• Spinal Muscular Atrophy Pipeline Therapies- OAV101, Risdiplam, Nusinersen, Amifampridine Phosphate, Vesemnogene Lantuparvovec, and others.
• Spinal Muscular Atrophy Therapeutic Assessment: Spinal Muscular Atrophy current marketed and emerging therapies
• Spinal Muscular Atrophy Market Dynamics: Attribute Analysis of Emerging Spinal Muscular Atrophy Drugs
• Spinal Muscular Atrophy Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Spinal Muscular Atrophy Unmet Needs, KOL's views, Analyst's views, Spinal Muscular Atrophy Market Access and Reimbursement

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Table of Content
1 Key Insights
2 Report Introduction
3 Spinal Muscular Atrophy Market Overview at a Glance
4 Spinal Muscular Atrophy Market: Methodology
5 Executive Summary of Spinal Muscular Atrophy
6 Key Events
7 Disease Background and Overview: Spinal Muscular Atrophy
8 Epidemiology and Patient Population
9 Patient Journey
10 Marketed Drugs
11 Emerging Drugs
12 Spinal Muscular Atrophy: Seven Major Market Analysis
13 KOL Views
14 SWOT Analysis
15 Unmet Needs
16 Market Access and Reimbursement
17 Appendix
18 DelveInsight Capabilities
19 Disclaimer

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