Spinal Muscular Atrophy (SMA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Spinal Muscular Atrophy (SMA) Market Outlook

Thelansis’s “Spinal Muscular Atrophy (SMA) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Spinal Muscular Atrophy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Spinal Muscular Atrophy (SMA) Overview

Spinal muscular atrophy (SMA) is characterized by the degeneration of alpha motor neurons in the anterior horn cells of the spinal cord, leading to progressive weakness and wasting of proximal muscles and, in severe cases, paralysis. The clinical presentation of SMA varies from severe to mild. It is classified into three subtypes: type I (also known as Werdnig Hoffmann disease), type II, and type III (also referred to as Kugelberg Welander disease), as well as type IV. This condition involves muscle weakness and atrophy due to progressive degeneration and irreversible loss of lower motor neurons in the spinal cord and brain stem nuclei. The onset of weakness can occur from prenatal stages to adulthood, typically displaying a symmetric, proximal > or distal pattern that worsens over time. Previously, SMA was categorized into clinical subtypes based on the maximum motor function achieved; however, it is now understood that the phenotype of SMN1-associated SMA exists along a continuum without clear subtype delineation. Common complications, even with supportive care, include poor weight gain leading to growth failure, restrictive lung disease, scoliosis, and joint contractures. Currently, there is no definitive cure for SMA, and treatment primarily focuses on symptom management and complication prevention. The U.S. Food and Drug Administration (FDA) has approved several medications for SMA treatment, including nusinersen (Spinraza™), onasemnogene abeparovec-xioi (Zolgensma™), and risdiplam (Evrysdi).

• The estimated prevalence of SMA in the USA is approximately 9 to 10 cases per 100,000 population, with an incidence ranging from 1.2 to 1.5 cases per 11,000 live births.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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