세계의 사지 근이영양증 치료 시장(2023-2030년)

Overview

The global limb girdle muscular dystrophy treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. A vast set of hereditary illnesses known as limb-girdle muscular dystrophies cause muscle loss and weakening (muscular dystrophy). To develop the illness (autosomal recessive inheritance), often both parents have to pass on the defective (inoperative) gene.

Government investments and research, particularly in developing economies, will continue to drive utilization of advanced technology for novel treatments such as gene therapy or corticosteroids for limb-girdle muscular dystrophy type 1 and type 2 and boost the global limb girdle muscular dystrophy treatment market.

Market Dynamics: Drivers & Restraints

Growing collaborations and regulatory approvals

Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by high investments, income levels, and infrastructure development. Several countries have experienced substantial demand for limb girdle muscular dystrophy treatment owing to the rising research. Various collaborations, acquisitions and license agreement with technological advancements among different countries will be a crucial factor driving the growth of the market.

On July 12, 2023, in order to create the first of its kind effective digital models for enhancing bioproduction produces in the area of gene therapy, Thales, a European developer in artificial intelligence for mission-critical systems, and Genethon, an innovator and leader in genetic therapy development and research for rare genetic diseases, are bringing together their expertise. Gene therapy treatment development is a very difficult, intricate, expensive, and time-consuming procedure. The reduction of production costs and time represents a significant challenge.

Additionally, on February 16, 2023, the limb-girdle muscular dystrophy (LGMD) medicine AB-1003 (also known as LION-101) has been given orphan drug designation by the European Commission (EC), according to Asklepios BioPharmaceutical, Inc., a fully owned and independently run subsidiary of Bayer AG.

Furthermore, the rising utilization of novel medicines and solutions for limb girdle muscular dystrophy. Rising muscular dystrophy cases and increasing utilization of several drugs along with research for innovative product development will be a major factor driving the growth of the limb girdle muscular dystrophy treatment market.

Side effects associated with the therapy

Although they are still relatively new forms of medicine, genetic treatments offer the potential to treat a wide range of illnesses. Certain cancers, allergic reactions, or organ or tissue damage if an injection is involved are examples of potential dangers. Genetic medicines are currently more safer owing to recent developments. Early research indicated very substantial health hazards associated with gene therapy, including toxicity, inflammation, and malignancy.

Segment Analysis

The global limb girdle muscular dystrophy treatment market is segmented based on type, treatment, end user and region.

Limb-girdle muscular dystrophy type 2 type segment accounted for approximately 56% of market share

Calpainopathy, also known as limb-girdle muscular dystrophy form 2A, is brought on by CAPN3 gene mutations. About 30% of cases of limb-girdle muscular dystrophy are Type 2A, which is also the most prevalent type. Dysferlinopathy is also known as limb-girdle muscular dystrophy form 2B and is brought on by DYSF gene mutations.

Initial symptoms of LGMD2A include frequently weakness and wastage (loss of muscular mass) in the shoulder, hip, and thigh muscles. Leg involvement is apparent earlier shoulder and arm involvement, and this weakness typically affects each side of the body equally.

On September 19, 2023, ATA-200, a gene therapy for type 2C/R5 limb-girdle muscular dystrophy (LGMD2C/R5) that is related to -sarcoglycan (SGCG), was the subject of a Clinical Trial Application (CTA) filed in Europe by Atamyo Therapeutics, a biotechnology company specializing in the creation of new-generation gene treatments targeting neuromuscular diseases. The business also said that ATA-200 has been approved for non-dilutive funding from France 2030, a public program run by Bpifrance, in the amount of up to €8 million ($8.6 million), to support their clinical trial and industrial development projects.

Geographical Penetration

North America accounted for around 39% of market share in 2022

Due to the rising need for limb girdle muscular dystrophy treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for limb girdle muscular dystrophy treatment.

Increasing expenditure on healthcare, advancement of technologies and different types of medications for several diseases, along with increase in biopharmaceutical or biotechnology business establishment across the region are also contributing to the growth of limb girdle muscular dystrophy treatment market share of this region. The market in this area is growing as people become more aware of various novel treatments. The aforementioned elements attest to North America's hegemonic position in the world.

North America continues to be a key player in the global limb girdle muscular dystrophy treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on rising advancements have fueled the demand for limb girdle muscle medications in the United States have been proactive in executing several initiatives or surgical procedures, stimulating these medications demand.

COVID-19 Impact Analysis

The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global limb girdle muscular dystrophy treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the pharmaceutical sector, with a significant consumer of corticosteroid or small molecule, was significantly impacted. The widespread lockdowns and restrictions imposed by the pandemic, which began in early 2020, had an effect on a number of initiatives all around the world.

Major medical and biotechnology industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for limb girdle muscular dystrophy treatment. Now several research studies have been initiated and companies have again started trial for their products efficiency. Overall, the impact of the pandemic on the global limb girdle muscular dystrophy treatment market is expected to be relatively moderate, with the market continuing to grow steadily due to the ongoing need and research for novel treatments.

Key Developments

• On August 3, 2023, the initial participant in the Phase 1 / Phase 2 LION-CS101 research study for people having limb-girdle muscular dystrophy subtype 2I/R9 (LGMD2I/R9) was recently dosed, according to Asklepios BioPharmaceutical, Inc., a genetic therapy company that is entirely owned and independently run as a subsidiary of Bayer AG. A brand-new FKRP replacement gene therapy under research is called AB-1003 (also referred to as LION-101).
• On June 22, 2023, the FDA accelerated the authorization of delandistrogene moxeparvovec-rokl (ELEVIDYS), an adeno-associated virus-based gene therapy, which is intended for the management of ambulatory children and adolescents with Duchenne muscular dystrophy (DMD) who have a confirmed mutation in the DMD gene, according to Sarepta Therapeutics, Inc., the pioneer in precision genetic therapy for rare diseases. Based on the levels of ELEVIDYS micro-dystrophin seen in ELEVIDYS-treated patients, this indication was given accelerated approval.

Competitive Landscape

The major global players in the market include: Jubilant Cadista Pharmaceuticals Inc., Sarepta Therapeutics, Inc., ML Bio Solutions, Genethon, Asklepios BioPharmaceutical, Inc., Atamyo Therapeutics, Vita Therapeutics, Inc., Edgewise Therapeutics, BridgeBio Inc. and Hansa Biopharma.

Why Purchase the Report?

• To visualize the global limb girdle muscular dystrophy treatment market segmentation based on type, treatment, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of limb girdle muscular dystrophy treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global limb girdle muscular dystrophy treatment market report would provide approximately 61 tables, 57 figures and 186 Pages.

Target Audience 2023

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

Table of Contents

1. Methodology and Scope

• 1.1. Research Methodology
• 1.2. Research Objective and Scope of the Report

2. Definition and Overview

3. Executive Summary

• 3.1. Snippet by Type
• 3.2. Snippet by Treatment
• 3.3. Snippet by End User
• 3.4. Snippet by Region

4. Dynamics

• 4.1. Impacting Factors
  • 4.1.1. Drivers
    • 4.1.1.1. Growing collaborations and regulatory approvals
    • 4.1.1.2. Rising technological advancements and developments
  • 4.1.2. Restraints
    • 4.1.2.1. Side effects associated with the therapy
  • 4.1.3. Opportunity
  • 4.1.4. Impact Analysis

5. Industry Analysis

• 5.1. Porter's Five Force Analysis
• 5.2. Supply Chain Analysis
• 5.3. Pricing Analysis
• 5.4. Regulatory Analysis
• 5.5. SWOT Analysis
• 5.6. Patent Analysis
• 5.7. Russia-Ukraine War Impact Analysis
• 5.8. DMI Opinion

6. COVID-19 Analysis

• 6.1. Analysis of COVID-19
  • 6.1.1. Scenario Before COVID
  • 6.1.2. Scenario During COVID
  • 6.1.3. Scenario Post COVID
• 6.2. Pricing Dynamics Amid COVID-19
• 6.3. Demand-Supply Spectrum
• 6.4. Government Initiatives Related to the Market During Pandemic
• 6.5. Manufacturers Strategic Initiatives
• 6.6. Conclusion

7. By Type

• 7.1. Introduction
  • 7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 7.1.2. Market Attractiveness Index, By Type
• 7.2. Limb-girdle Muscular Dystrophy Type 1*
  • 7.2.1. Introduction
  • 7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 7.3. Limb-girdle Muscular Dystrophy Type 2

8. By Treatment

• 8.1. Introduction
  • 8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 8.1.2. Market Attractiveness Index, By Treatment
• 8.2. Medications *
  • 8.2.1. Introduction
  • 8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
  • 8.2.3. Corticosteroids
    • 8.2.3.1. Prednisone
    • 8.2.3.2. Deflazacort (Emflaza)
  • 8.2.4. Small Molecule
    • 8.2.4.1. Ribitol (BBP-418)
• 8.3. Gene Therapy
• 8.4. Others

9. By End User

• 9.1. Introduction
  • 9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 9.1.2. Market Attractiveness Index, By End User
• 9.2. Hospitals*
  • 9.2.1. Introduction
  • 9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
• 9.3. Ambulatory Surgical Centers
• 9.4. Others

10. By Region

• 10.1. Introduction
  • 10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
  • 10.1.2. Market Attractiveness Index, By Region
• 10.2. North America
  • 10.2.1. Introduction
  • 10.2.2. Key Region-Specific Dynamics
  • 10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.2.6.1. U.S.
    • 10.2.6.2. Canada
    • 10.2.6.3. Mexico
• 10.3. Europe
  • 10.3.1. Introduction
  • 10.3.2. Key Region-Specific Dynamics
  • 10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.3.6.1. Germany
    • 10.3.6.2. UK
    • 10.3.6.3. France
    • 10.3.6.4. Italy
    • 10.3.6.5. Spain
    • 10.3.6.6. Rest of Europe
• 10.4. South America
  • 10.4.1. Introduction
  • 10.4.2. Key Region-Specific Dynamics
  • 10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.4.6.1. Brazil
    • 10.4.6.2. Argentina
    • 10.4.6.3. Rest of South America
• 10.5. Asia-Pacific
  • 10.5.1. Introduction
  • 10.5.2. Key Region-Specific Dynamics
  • 10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
  • 10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
    • 10.5.6.1. China
    • 10.5.6.2. India
    • 10.5.6.3. Japan
    • 10.5.6.4. Australia
    • 10.5.6.5. Rest of Asia-Pacific
• 10.6. Middle East and Africa
  • 10.6.1. Introduction
  • 10.6.2. Key Region-Specific Dynamics
  • 10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Type
  • 10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
  • 10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User

11. Competitive Landscape

• 11.1. Competitive Scenario
• 11.2. Market Positioning/Share Analysis
• 11.3. Mergers and Acquisitions Analysis

12. Company Profiles

• 12.1. Jubilant Cadista Pharmaceuticals Inc.*
  • 12.1.1. Company Overview
  • 12.1.2. Product Portfolio and Description
  • 12.1.3. Financial Overview
  • 12.1.4. Key Developments
• 12.2. Sarepta Therapeutics, Inc.
• 12.3. ML Bio Solutions
• 12.4. Genethon
• 12.5. Asklepios BioPharmaceutical, Inc.
• 12.6. Atamyo Therapeutics
• 12.7. Vita Therapeutics, Inc.
• 12.8. Edgewise Therapeutics
• 12.9. BridgeBio Inc.
• 12.10. Hansa Biopharma

LIST NOT EXHAUSTIVE

13. Appendix

• 13.1. About Us and Services
• 13.2. Contact Us