US Spinal Muscular Atrophy (SMA) Therapeutics Market Report 2023 to 2030

US Spinal Muscular Atrophy (SMA) Therapeutics Market Analysis

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US Spinal Muscular Atrophy (SMA) Therapeutics Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 ? 2030. The market is expanding as a result of rising SMA instances, technological advancements, government encouragement, rising consumer awareness, and increased R&D activities. Major global players in Spinal Muscular Atrophy (SMA) Therapeutics Market are Sanofi (France), F. Hoffmann-La Roche Ltd. (Switzerland), Boehringer Ingelheim International GmbH. (Germany), GSK plc (U.K.), Novartis AG (Switzerland), Biogen (U.S.), Pfizer Inc.(U.S.), Ionis Pharmaceuticals, Inc. (U.S.), Regeneron Pharmaceuticals Inc (U.S.)., Abbott (U.S.), AbbVie Inc. (U.S.), Novo Nordisk A/S (Denmark), Takeda Pharmaceutical Company Limited (Japan), AstraZeneca (U.K.), Leadiant Biosciences, Inc.(Italy), Catalyst Pharmaceuticals, Inc.(U.S.), PTC Therapeutics (U.S.)

ID: IN10USRD067 CATEGORY: Rare Diseases GEOGRAPHY: US AUTHOR: Gaurav Bhongale

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US Spinal Muscular Atrophy (SMA) Therapeutics Market Analysis Summary

US Spinal Muscular Atrophy (SMA) Therapeutics Market is valued at around $1.95 Bn in 2022 and is projected to reach $6.87 Bn by 2030, exhibiting a CAGR of 17.1% during the forecast period 2023-2030.

Spinal muscular atrophy is a neuromuscular condition that causes muscles to deteriorate (SMA). People with SMA lose their motor neurons, a particular class of nerve cells in the spinal cord that controls muscular movement. The nerve signals that drive muscles to contract cannot reach the muscles without these motor neurons. Atrophy is a medical term that denotes reduction. Several muscles in SMA atrophy and shrink due to lack of use. Between 10,000 and 25,000 children and adults in the US have SMA. It affects one in every 6,000 to 10,000 children. SMA is brought on by a missing or altered (mutated) portion of the SMN1 gene. The SMN protein is created by the SMN1 gene. This protein is necessary for the survival and efficient operation of motor neurons. Due to insufficient SMN protein production, motor neurons in SMA patients deteriorate and eventually die. This makes it impossible for the brain to control voluntary actions, especially those involving the head, neck, arms, and legs.

The market is expanding as a result of rising SMA instances, technological advancements, government encouragement, rising consumer awareness, and increased R&D activities. The market is growing due to the increasing popularity of spinraza. Both paediatric and adult patients are treated with a prescription medication called Spinraza. The intrathecal injection is used by trained healthcare personnel to give it. On a global scale, a number of countries, including Norway, Italy, the Netherlands, Croatia, and Poland, have entirely covered the cost of Spinraza. The market is able to grow as a result of this. Major global players in Spinal Muscular Atrophy (SMA) Therapeutics Market are Sanofi (France), F. Hoffmann-La Roche Ltd. (Switzerland), and Boehringer Ingelheim International GmbH. (Germany), GSK plc (U.K.), Novartis AG (Switzerland), Biogen (U.S.), Pfizer Inc.(U.S.), Ionis Pharmaceuticals, Inc. (U.S.), Regeneron Pharmaceuticals Inc (U.S.)., Abbott (U.S.), AbbVie Inc. (U.S.), Novo Nordisk A/S (Denmark), Takeda Pharmaceutical Company Limited (Japan), AstraZeneca (U.K.), Leadiant Biosciences, Inc.(Italy), Catalyst Pharmaceuticals, Inc.(U.S.), PTC Therapeutics (U.S.).

Market Dynamics

Market Drivers

Increasing Knowledge of SMA's Novel Therapies

The growing acceptability of novel therapies is one of the main reasons for promoting market progress. For instance, Cure SMA organised the 2022 Annual SMA Conference in Anaheim in 2021 to bring together the SMA community, which includes patients, researchers, their caregivers, and clinicians. In addition to this conference, a number of other occasions were hosted, including social gatherings and instructional programmes. Manufacturers have the opportunity to talk with SMA patients at these events and learn more about their unmet medical needs. The market's growth is accelerated by this factor.

Increasing Spinraza Demand

The market is growing because of Spinraza's increasing popularity. Adult and paediatric patients are also treated with the prescription medication Spinraza. The intrathecal injection is used to give it by trained medical personnel. The cost of Spinraza has been fully reimbursed by a number of countries, including Norway, Italy, the Netherlands, Croatia, and Poland. Because of this, the market is growing.

Market Developments

Scholar rocks’ Apitegromab is a fully human anti-proMyostatin monoclonal antibody (mAb) of the immunoglobulin G4 (IgG4)/lambda isotype that specifically binds to human pro/latent myostatin with high affinity inhibiting myostatin activation. Its Phase 3 trial (Study SRK-015-003) is being conducted in patients ≥2 years old at Screening, who were previously diagnosed with later-onset spinal muscular atrophy (SMA) estimated to get completed by 2024.
On 14 March 2023, Biohaven Pharmaceuticals announced that they have received Fast Track designation from the U.S. Food and Drug Administration (FDA) for Taldefgrobep alfa, a protein designed to inhibit the function of myostatin to increase muscle size and function which can be used in SMA

Key players

Biogen Inc. Novartis AG Roche Holding AG AveXis, Inc. Cytokinetics, Incorporated Scholar Rock Holding Corporation PTC Therapeutics, Inc. Ionis Pharmaceuticals, Inc. Regenxbio Inc. Astellas Pharma Inc.

1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country

2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)

3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints

4. Competitive Landscape
4.1 Major Market Share

4.2 Key Company Profile (Check all Companies in the Summary Section)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 Financials (Based on Availability)

5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment

6. Methodology and Scope

Market Segmentations For US Spinal Muscular Atrophy (SMA) Therapeutics Market

By Type

Werdnig-Hoffmann Disease
Infant SMA
Kugelberg-Welander Disease
Adult SMA

By Treatment

Medication
Physical Therapy
Surgery
Others

By Route of Administration

Intrathecal
Intraspinal
Subcutaneous
Others

Methodology for Database Creation

Our database offers a comprehensive list of healthcare centers, meticulously curated to provide detailed information on a wide range of specialties and services. It includes top-tier hospitals, clinics, and diagnostic facilities across 30 countries and 24 specialties, ensuring users can find the healthcare services they need.

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How Do We Get It?

Our database is created and maintained through a combination of secondary and primary research methodologies.

1. Secondary Research

With many years of experience in the healthcare field, we have our own rich proprietary data from various past projects. This historical data serves as the foundation for our database. Our continuous process of gathering data involves:

Analyzing historical proprietary data collected from multiple projects.
Regularly updating our existing data sets with new findings and trends.
Ensuring data consistency and accuracy through rigorous validation processes.

With extensive experience in the field, we have developed a proprietary GenAI-based technology that is uniquely tailored to our organization. This advanced technology enables us to scan a wide array of relevant information sources across the internet. Our data-gathering process includes:

Searching through academic conferences, published research, citations, and social media platforms
Collecting and compiling diverse data to build a comprehensive and detailed database
Continuously updating our database with new information to ensure its relevance and accuracy

2. Primary Research

To complement and validate our secondary data, we engage in primary research through local tie-ups and partnerships. This process involves:

Collaborating with local healthcare providers, hospitals, and clinics to gather real-time data.
Conducting surveys, interviews, and field studies to collect fresh data directly from the source.
Continuously refreshing our database to ensure that the information remains current and reliable.
Validating secondary data through cross-referencing with primary data to ensure accuracy and relevance.

Combining Secondary and Primary Research

By integrating both secondary and primary research methodologies, we ensure that our database is comprehensive, accurate, and up-to-date. The combined process involves:

Merging historical data from secondary research with real-time data from primary research.
Conducting thorough data validation and cleansing to remove inconsistencies and errors.
Organizing data into a structured format that is easily accessible and usable for various applications.
Continuously monitoring and updating the database to reflect the latest developments and trends in the healthcare field.

Through this meticulous process, we create a final database tailored to each region and domain within the healthcare industry. This approach ensures that our clients receive reliable and relevant data, empowering them to make informed decisions and drive innovation in their respective fields.