DelveInsight’s “Spinal Muscular Atrophy (SMA) Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Spinal Muscular Atrophy market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The report covers emerging Spinal Muscular Atrophy drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Spinal Muscular Atrophy treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Spinal Muscular Atrophy (SMA): An Overview
Spinal muscular atrophy (SMA) is an autosomal recessive rare neuromuscular disorder that is characterized by progressive muscle wasting because of the loss of motor neurons in the spinal cord (anterior horn), which controls voluntary muscle movement. SMA is one of the most common causes of infant death and mobility impairment. It mostly affects proximal muscles and respiratory muscles. The most common form of SMA is known as ‘5q SMA’ due to its genetic cause.
SMA follows the autosomal mode of inheritance, and its onset ranges from infancy to adolescence. Human chromosome 5q has two identical genes, SMN1 and SMN2. SMN1 encodes 100% fully functional SMN protein, whereas SMN2 encodes only 10-20% of functional SMN protein. Mutation of SMN1 is the main reason for this disorder, and the severity of the disease depends on the number of SMN2 gene copies. The healthy person carries 2 copies of the SMN2 gene, but in SMA-affected patients, it can vary from 1 to 4. The greater the number of SMN2 gene copies, the milder the effect of the disorder. The other SMAs form a genetically very heterogeneous group of rare disorders, differing in their mode of inheritance, the topography of the muscular deficit, and the association with other neurological abnormalities.
The FDA-approved therapies for SMA include Spinraza, Evrysdi, and a one-time gene therapy, Zolgensma. Moreover, the management of SMA is based on supportive and multidisciplinary care with a focus on reducing complications and improving quality of life.
Spinal Muscular Atrophy Market Key Facts
According to the National Organization for Rare Disorders (NORD), the prevalence of all types of SMA has been estimated to be 4-7.8 per 100,000 live births. Approximately 80% of SMA patients have the Werdnig-Hoffmann form, i.e. Type 1 SMA. It further stated that the condition is diagnosed in only 150 to 200 people. Moreover, 1 in 50 people (approximately 6 million Americans) are carriers.
National Institute for Health and Care Excellence (NICE) (2018) reported that SMA affects an estimated 1 in 6,000 to 1 in 10,000 births worldwide. It is estimated that about 100 people are born with SMA per year in the UK, and there are currently between 1,200 and 2,500 children and adults in the UK living with SMA.
A study conducted by Ghetti et al. (2020) stated that currently, the estimated number of SMA patients in Italy is 850, corresponding to a prevalence of 1.41 per 100,000 inhabitants. This number, confirmed by patient associations, is likely underestimated due to the lack of follow-up of patients with less severe forms.
The total prevalent population of SMA in the 7MM was around 20,000 cases in 2020, out of which the United States accounted for nearly 13,000 cases in 2020.
Spinal Muscular Atrophy Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Spinal Muscular Atrophy pipeline therapies. It also thoroughly assesses the Spinal Muscular Atrophy market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete details of the market trend for each marketed Spinal Muscular Atrophy drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Spinal Muscular Atrophy Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Spinal Muscular Atrophy epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Spinal Muscular Atrophy epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.
The Report Covers the Spinal Muscular Atrophy Epidemiology, Segmented as –
Total Prevalent Population of Spinal Muscular Atrophy (SMA) in the 7MM [2019-2032]
Age-specific Prevalence of Spinal Muscular Atrophy (SMA) in the 7MM [2019-2032]
Type-specific Prevalence of Spinal Muscular Atrophy (SMA) in the 7MM [2019-2032]
Spinal Muscular Atrophy Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Spinal Muscular Atrophy market or expected to be launched during the study period. The analysis covers the Spinal Muscular Atrophy market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Spinal Muscular Atrophy drugs based on their sale and market share.
The report also covers the Spinal Muscular Atrophy pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Spinal Muscular Atrophy companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Spinal Muscular Atrophy Therapeutics Analysis
With the approval of three therapies for SMA (Spinraza, Evrysdi, and Zolgensma (gene therapy)), some of th unmet needs for the treatment of SMA were fulfilled. Moreover, the management of SMA is also based on supportive and multidisciplinary care with a focus on reducing complications and improving quality of life. The current market holds alternatives for symptomatic relief. However, approaches for SMA management also focus on nutrition, respiratory care assessment, respiratory muscle weakness, orthopedic care, rehabilitation, and a few off-label medications.
Treatment of SMA is quite challenging however, in an overall perspective, recent, and extensive research and development activities being conducted for the development of novel drugs, increased understanding of the related comorbidities, along with the upcoming pipeline drugs, is expected to create a lucrative environment for growth of SMA treatment market. To further improve the treatment scenario, several major pharma and biotech companies are developing therapies for Spinal muscular atrophy. Currently, Scholar Rock is leading the therapeutics market with its Spinal muscular atrophy drug candidates in the most advanced stage of clinical development.
Spinal Muscular Atrophy Companies Actively Working in the Therapeutics Market Include
Scholar Rock
Biogen
Astellas Pharma
Alcyone Therapeutics
AndroScience Corporation
Hanugen Therapeutics
Voyager Therapeutics
Hoffmann-La Roche
Catalyst Pharmaceuticals
NMD Pharma
Biohaven Pharmaceuticals
CANbridge Pharmaceuticals Inc.
Aurimed Pharma
Exicure
Amylon Therapeutics
Amniotics
And Many More
Emerging and Marketed Spinal Muscular Atrophy Therapies Covered in the Report Include:
Apitegromab: Scholar Rock
ACTX-401: Alcyone Therapeutics
Spinraza (nusinersen): Biogen
Reldesemtiv: Cytokinetics
GYM329: Roche
Zolgensma: Novartis
Evrysdi: Hoffmann-La Roche
And Many Others
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Spinal Muscular Atrophy Competitive Intelligence Analysis
4. Spinal Muscular Atrophy Market Overview at a Glance
5. Spinal Muscular Atrophy Disease Background and Overview
6. Spinal Muscular Atrophy Patient Journey
7. Spinal Muscular Atrophy Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Spinal Muscular Atrophy Treatment Algorithm, Current Treatment, and Medical Practices
9. Spinal Muscular Atrophy Unmet Needs
10. Key Endpoints of Spinal Muscular Atrophy Treatment
11. Spinal Muscular Atrophy Marketed Therapies
12. Spinal Muscular Atrophy Emerging Drugs and Latest Therapeutic Advances
13. Spinal Muscular Atrophy Seven Major Market Analysis
14. Attribute Analysis
15. Spinal Muscular Atrophy Market Outlook (In US, EU5, and Japan)
16. Spinal Muscular Atrophy Companies Active in the Market
17. Spinal Muscular Atrophy Access and Reimbursement Overview
18. KOL Views on the Spinal Muscular Atrophy Market
19. Spinal Muscular Atrophy Market Drivers
20. Spinal Muscular Atrophy Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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