Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast - 2028
発行: DelveInsight Business Research LLP
ページ情報: 英文 170 Pages
DelveInsight's 'Spinal Muscular Atrophy (SMA) - Market Insights, Epidemiology and Market Forecast-2028' report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Spinal Muscular Atrophy in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.
The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Spinal Muscular Atrophy from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.
Study Period: 2017-2028
According to the National Institute of Health (NIH), Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting (atrophy) in muscles used for movement (skeletal muscles). It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement. The weakness tends to be more severe in the muscles that are close to the center of the body (proximal) compared to muscles away from the body's center (distal). The muscle weakness usually worsens with age. SMA is one of the most common causes of infant death and mobility impairment. It mostly affects proximal muscles and respiratory muscles. There are three common forms of spinal muscular atrophy classified by age at onset and severity of functional impairment. Type 1 spinal muscular atrophy has been termed Werdnig-Hoffmann disease and is most usually fatal by 2 years of age. Children with spinal muscular atrophy type 2 can usually achieve the ability to sit, but have limited walking. Patients with type 3 or Kugelberg-Welander syndrome develop weakness in teen or young adult years.
The DelveInsight Spinal Muscular Atrophy market report gives the thorough understanding of the Spinal Muscular Atrophy by including details such as disease introduction, classification, causes, pathophysiology, biomarkers in Spinal Muscular Atrophy, symptoms, diagnosis and differential diagnosis. It also provides treatment algorithms and treatment guidelines for Spinal Muscular Atrophy in the US, Europe, and Japan.
The Spinal Muscular Atrophy epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalent Cases of Spinal Muscular Atrophy, Total Diagnosed Prevalent Cases of Spinal Muscular Atrophy, Type specific Diagnosed Prevalence of Spinal Muscular Atrophy, Mutation specific Prevalence of Spinal Muscular Atrophy) scenario of Spinal Muscular Atrophy in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2028.
According to a study conducted by R. Vamshi et al titled as "Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease" published in Journal of Managed Care and Specialty Pharmacy (JMCP) which stated the SMN1 gene product-SMN protein-is crucial for motor neuron development. In approximately 95% of patients, SMA results from homozygous deletion or conversion of SMN1. In about 2% of patients, de novo deletions occur in one of the SMN1 alleles; in 3%-4%, other mutations can be found, typically with an SMN1 deletion on the other allele.
This segment of the Spinal Muscular Atrophy report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
Current management of SMA is based on supportive and multidisciplinary care with a focus on reducing complications and improving quality of life. Pulmonary disease is the major cause of mortality in SMA type I and II and affects a small proportion of patients with SMA type III. Management strategies include airway clearance, cough assistance, nocturnal non-invasive ventilatory support, and continuous non-invasive ventilation. The supportive approaches involve nutrition, orthopedic care, rehabilitation, long-term feeding tubes etc.
Spinraza, manufactured by Biogen is the only approved drug for the treatment of Spinal Muscular Atrophy in adults and children. The active ingredient of the drug is Nusinersen. The drug increases full-length survival motor neuron (SMN) protein by targeting the process through which it is produced by the SMN2 gene.
There is no cure, but treatments can improve some symptoms and in some cases, help the patients live longer. Researchers are working robustly to find new ways to fight the disease.
To meet the increasing demand for the treatment of Spinal Muscular Atrophy, companies have shifted their focus towards the development of targeted therapies. Expected launch of potential therapies may increase the market size in the coming years, assisted by an increase in the Incident population of Spinal Muscular Atrophy& awareness of the disease. The overall dynamics of Spinal Muscular Atrophy market is anticipated to change in the coming years owing to the expected launch of emerging therapies of the major key players such as Novartis, Hoffmann-La Roche, Astellas Pharma, Cytokinetics, and some others, will significantly increase the market during the forecast period (2019-2028).
The Spinal Muscular Atrophy market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a thorough detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the market of Spinal Muscular Atrophy in 7MM is expected to change from 2019-2028.
This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Expected launch of therapies for Spinal Muscular Atrophy such as Zolgensama (Novartis), Risdiplam (Astellas Pharma/Cytokinetics), Branaplam (Novartis) and other targeted therapies in the forecast period [2019-2028] will also create a positive impact on the Spinal Muscular Atrophy market.