New York, USA, Oct. 31, 2023 (GLOBE NEWSWIRE) -- Spinal Muscular Atrophy Market to Grow Rapidly During the Forecast Period (2019–2032), Assesses DelveInsight | Key Players - Biogen, NMD Pharma, Novartis, Roche, Biohaven Pharmaceuticals, Scholar Rock, AnnJi Pharmaceutical
The dynamics of the spinal muscular atrophy market are anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, rising awareness of the disease, incremental healthcare spending across the world, and the expected launch of emerging therapies during the forecast period.
DelveInsight’s Spinal Muscular Atrophy Market Insights report includes a comprehensive understanding of current treatment practices, spinal muscular atrophy emerging drugs, market share of individual therapies, and current and forecasted spinal muscular atrophy market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Key Takeaways from the Spinal Muscular Atrophy Market Report
- As per DelveInsight’s analysis, the spinal muscular atrophy market is anticipated to grow at a significant CAGR by 2032.
- As per Delveinsight’s analysis, the total prevalent population of SMA in the 7MM was around 20K cases in 2020, out of which the United States accounted for nearly 13K cases in 2020.
- Leading spinal muscular atrophy companies such as Biogen, NMD Pharma A/S, Novartis Pharmaceuticals, Hoffmann-La Roche, Biohaven Pharmaceuticals, Inc., Scholar Rock, Inc., AnnJi Pharmaceutical Co., Ltd., and others are developing novel spinal muscular atrophy drugs that can be available in the spinal muscular atrophy market in the coming years.
- Some of the key therapies for spinal muscular atrophy treatment include Nusinersen, NMD670, OAV101, Risdiplam, Taldefgrobep alfa, SRK-015, AJ201, and others.
- The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021.
- The US FDA has granted orphan drug designation and the European Medicines Agency (EMA) has granted orphan medicinal product designation to reldesemtiv for the potential treatment of SMA.
Discover which therapies are expected to grab the major spinal muscular atrophy market share @ Spinal Muscular Atrophy Market Report
Spinal Muscular Atrophy Overview
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that follows an autosomal recessive inheritance pattern. It is characterized by progressive muscle wasting resulting from the degeneration of motor neurons in the anterior horn of the spinal cord, which controls voluntary muscle movement. SMA is a leading cause of infant mortality and mobility impairment, primarily affecting proximal and respiratory muscles. The most prevalent form of SMA is referred to as ‘5q SMA’ due to its genetic origin. The severity of symptoms can vary widely depending on the type of SMA and its onset. In general, individuals with SMA may experience muscle weakness, muscle atrophy (shrinkage), and decreased motor function. Common symptoms include difficulty with tasks like crawling, walking, and lifting objects. In more severe cases, SMA can lead to respiratory difficulties, difficulty swallowing, and compromised overall mobility. SMA diagnosis typically involves a physical examination, family history assessment, and a range of diagnostic tests, including creatine kinase (CK) level testing, muscle biopsy, prenatal testing, newborn screening, among others.
Spinal Muscular Atrophy Epidemiology Segmentation
The spinal muscular atrophy epidemiology section provides insights into the historical and current spinal muscular atrophy patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders.
The spinal muscular atrophy market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
- Total Spinal Muscular Atrophy Prevalent Cases
- Spinal Muscular Atrophy Type-specific Prevalent Cases
- Spinal Muscular Atrophy Age-specific Prevalent Cases
Download the report to understand which factors are driving spinal muscular atrophy epidemiology trends @ Spinal Muscular Atrophy Epidemiological Insights
Spinal Muscular Atrophy Treatment Market
The introduction of three approved therapies for SMA (Spinraza, Evrysdi, and Zolgensma, a gene therapy) has addressed several unmet needs in SMA treatment. Additionally, managing SMA involves a comprehensive approach that includes supportive and multidisciplinary care aimed at minimizing complications and enhancing the quality of life. The current market also offers options for symptomatic relief. Furthermore, SMA management strategies encompass aspects such as nutrition, respiratory care assessment, addressing respiratory muscle weakness, orthopedic care, rehabilitation, and the use of some off-label medications.
Novartis’ Zolgensma, an adeno-associated virus vector-based gene therapy, is prescribed for the treatment of spinal muscular atrophy in children under 2 years old with bi-allelic SMN1 gene mutations. The primary goal of this therapy is to introduce a functional human SMN gene into targeted motor neuron cells, thereby stimulating the production of sufficient SMN protein to enhance motor neuron function and maintain consistent protein expression.
Evrysdi, developed by Hoffmann-La Roche, is a splicing modifier targeting survival motor neuron 2 (SMN2). This medication is prescribed for the treatment of spinal muscular atrophy (SMA) in patients 2 months of age and older, offering the convenience of daily administration by mouth or feeding tube in a home setting. It is a breakthrough therapy for SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency.
Biogen’s drug Spinraza has gained approval for the treatment of spinal muscular atrophy (SMA) in both pediatric and adult patients. Nusinersen serves as the active ingredient within this medication. It’s worth noting that Nusinersen is a modified oligonucleotide, where the 2’-hydroxyl groups of the ribofuranosyl rings have been replaced by 2’-O-2-methoxyethyl groups, and the phosphate linkages have been substituted with phosphorothioate linkages. The specific target of Nusinersen is the intronic splicing silencer N1 (ISS-N1) found in exon 7 of the SMN2 transcript.
Learn more about the FDA-approved drugs for spinal muscular atrophy @ Drugs for Spinal Muscular Atrophy Treatment
Key Spinal Muscular Atrophy Therapies and Companies
- Nusinersen: Biogen
- NMD670: NMD Pharma A/S
- OAV101: Novartis Pharmaceuticals
- Risdiplam: Hoffmann-La Roche
- Taldefgrobep alfa: Biohaven Pharmaceuticals, Inc.
- SRK-015: Scholar Rock, Inc.
- AJ201: AnnJi Pharmaceutical Co., Ltd.
To know more about spinal muscular atrophy clinical trials, visit @ Spinal Muscular Atrophy Treatment Drugs
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. Evrysdi represents an orally administered SMA medication, offering the convenience of at-home treatment and potentially being a preferred choice over Spinraza, which requires intrathecal administration. Currently, there are promising SMA drugs in mid- and early-phase development, spearheaded by Roche, Scholar Rock, and Cytokinetics. It is imperative for other pharmaceutical companies to contribute to the development of therapies that cater to the diagnostic and medical requirements of less common SMA variants, given the significant market potential.
However, several factors may impede the growth of the spinal muscular atrophy market. Research indicates that some SMA patients treated with the gene therapy Zolgensma may require additional treatment, as not all motor neurons are effectively transduced by the gene therapy, leaving certain neurons untreated. Unfortunately, the high cost of Zolgensma makes it nearly unaffordable for the general population. Furthermore, the annual expenses associated with Spinraza and Evrysdi therapy are also significantly elevated, placing a heavy financial burden on patients.
| Spinal Muscular Atrophy Report Metrics | Details |
| Study Period | 2019–2032 |
| Spinal Muscular Atrophy Report Coverage | 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] |
| Key Spinal Muscular Atrophy Companies | Biogen, NMD Pharma A/S, Novartis Pharmaceuticals, Hoffmann-La Roche, Biohaven Pharmaceuticals, Inc., Scholar Rock, Inc., AnnJi Pharmaceutical Co., Ltd., and others |
| Key Spinal Muscular Atrophy Therapies | Nusinersen, NMD670, OAV101, Risdiplam, Taldefgrobep alfa, SRK-015, AJ201, and others |
Scope of the Spinal Muscular Atrophy Market Report
- Therapeutic Assessment: Spinal Muscular Atrophy current marketed and emerging therapies
- Spinal Muscular Atrophy Market Dynamics: Attribute Analysis of Emerging Spinal Muscular Atrophy Drugs
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Spinal Muscular Atrophy Market Access and Reimbursement
Discover more about spinal muscular atrophy drugs in development @ Spinal Muscular Atrophy Clinical Trials
Table of Contents
| 1. | Spinal Muscular Atrophy Market Key Insights |
| 2. | Spinal Muscular Atrophy Market Report Introduction |
| 3. | Spinal Muscular Atrophy Market Overview at a Glance |
| 4. | Spinal Muscular Atrophy Market Executive Summary |
| 5. | Disease Background and Overview |
| 6. | Spinal Muscular Atrophy Treatment and Management |
| 7. | Spinal Muscular Atrophy Epidemiology and Patient Population |
| 8. | Patient Journey |
| 9. | Spinal Muscular Atrophy Marketed Drugs |
| 10. | Spinal Muscular Atrophy Emerging Drugs |
| 11. | Seven Major Spinal Muscular Atrophy Market Analysis |
| 12. | Spinal Muscular Atrophy Market Outlook |
| 13. | Potential of Current and Emerging Therapies |
| 14. | KOL Views |
| 15. | Unmet Needs |
| 16. | SWOT Analysis |
| 17. | Appendix |
| 18. | DelveInsight Capabilities |
| 19. | Disclaimer |
| 20. | About DelveInsight |
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