Japan Spinal Muscular Atrophy (SMA) Therapeutics Market Analysis

Japan Spinal Muscular Atrophy (SMA) Therapeutics Market Analysis


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Japan 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: IN10JPRD067 CATEGORY: Rare Diseases GEOGRAPHY: Japan AUTHOR: Nipun Arora

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Japan Spinal Muscular Atrophy (SMA) Therapeutics Market Analysis Summary

Japan Spinal Muscular Atrophy (SMA) Therapeutics Market is valued at around $0.29 Bn in 2022 and is projected to reach $1.21 Bn by 2030, exhibiting a CAGR of 19.6% 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

Avexis Biogen Novartis Pfizer Sanofi PTC Therapeutics Inc. Orchard Therapeutics plc uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals

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 Japan 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

Insights10 will provide you with the reports within 10 key parameters which are:

  1. Market Overview
  2. Market Growth Drivers & Restraints
  3. Epidemiology of Disease Type
  4. Market Segmentation
  5. Market Share
  6. Competitive Landscape
  7. Key Company Profiles
  8. Healthcare Policies & Regulatory Framework
  9. Reimbursement Scenario
  10. Factors Driving Future Growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision-making for business stakeholders. Our focused approach to developing reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

 

Stage I: Market Data Collection

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Market Trends: We generally look at macro parameters and micro indicators. The macro parameters include changes in government policies, demand and supply of the market, government intervention programs, and major market share. The micro indicators are GDP growth, market size, market volume, etc. We also understand nuances specific to each country like the US, Canada, India, Germany, etc., and have worked across 60+ countries and hence not only understand global trends but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

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The study on the market covers the analysis of the leading geographies such as Asia-Pacific, Africa, Europe, Middle East, North America, and  Latin America for the period of 2022 to 2030. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape, and policy and regulatory scenario, and the quantitative analysis covers different market estimates and forecasts.

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We make sure to finalize the numbers in alignment with the market research. Firstly, our internal experts ensure thorough validation and checking to ensure accurate and precise analysis and then validation is also done using a multiple-data analysis model. Two-level validation is done and entails the finalization of the report scope and the way of representation pattern.

 

 

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analyzed and synthesized. The second series of interviews are done if necessary to check and validate. The future opportunities are analyzed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results is then, interpreted by considering the following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing the regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue/volume
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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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Last updated on: 04 July 2023
Updated by: Anish Swaminathan

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