Mitochondrial Myopathy Diagnosis & Treatment Market research Report - Global Forecast till 2027

Global Mitochondrial Myopathy Diagnosis & Treatment Market Research Report: Information by Type (Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-Like Episodes, Progressive External Ophthalmoplegia, Myoclonic Epilepsy with Ragged Red Fibers, Mitochondrial Neurogastrointestinal Encephalopathy Syndrome, Kearns–Sayre Syndrome (KSS), Mitochondrial DNA Depletion Syndrome, Pearson Syndrome, Leigh Syndrome, Neuropathy, Ataxia and others), Diagnostic Test (Genetic Tests, Muscle Biopsy and Biochemical Test), Deployment Models (On-Premise and Cloud-Based), Therapy (Supportive Therapy and Targeted Therapy) and Region - Forecast Till 2027

ID: MRFR/Pharma/2648-CR | July 2019 | Region: Global | 61 pages

Please note that the assessment period of report has been updated from 2017-2025 to 2020-2027. Cordially fill the sample form for updated data.

Market Scenario


The Global Mitochondrial Myopathy Diagnosis & Treatment Market is growing at a healthy pace and is expected to be USD 20.4 million in 2016, at a CAGR of 9.8% during the forecast period 2017-2025.


The mitochondrial myopathy treatment provides relief from mitochondrial myopathies such as Barth syndrome, chronic progressive external ophthalmoplegia (CPEO), Kearns-Sayre syndrome (KSS), etc. There exists no cure for the mitochondrial myopathy, although many drugs are in the pipeline, and few have received fast track approval and orphan drug status. The factors such as rising number of screening services for mitochondrial disorders, a growing number of mitochondrial myopathy cases, increased government funding, and incentives for rare genetic diseases are driving the market for mitochondrial myopathy diagnosis & treatment. However, inadequate treatment options and the absence of curative treatment have constrained the market. The treatment options have been divided into curative and symptomatic management of conditions associated with mitochondrial myopathies such as stroke and cardiac symptoms.


Market Dynamics


Mitochondrial myopathy is a rare mutational disease, and at present, there is no concrete treatment available for the disease. However, there are few diagnostic tests available in the market, which are the only saving grace for the global mitochondrial myopathy diagnosis and treatment market at present. There are certain factors which drive the growth of the market. These include a rise in the number of mitochondrial myopathy patients, development in the disease screening process and testing services, lucrative incentives for the rare genetic diseases, and government funding and support for the development of the treatment options.


Further, rising number of screening and a growing number of mitochondrial myopathy casesfor example, in the US, new-born screening is performed on every infant regardless of the parent’s health insurance status or paying ability with some states not even charging the nominal fees for the testing. Any other expenses are covered by most private health insurance plans and the Children's Health Insurance Program (CHIP) and Medicaid both cover the cost. Thus, rising screening both due to government regulations and increasing insurance penetration is estimated to drive the future growth of mitochondrial myopathies treatment market.


On the other hand, poor treatment options and unavailability of the curative treatment for the mitochondrial myopathy, lack of awareness, and a high number of the underdiagnosed population are some of the factors which hinder the growth of the mitochondrial myopathy diagnosis & treatment market.


Figure 1: - Global Mitochondrial Myopathy Diagnosis & Treatment Share, by Region 2017 (%)  Mitochondrial Myopathy Diagnosis & Treatment


Sources: WHO, Centers for Medicare & Medicaid Services, Annual Reports, Press Release, White Paper, and Company Presentation


Segmentation


The global mitochondrial myopathy diagnosis & treatment market has been segmented based on type, services, diagnostic test, therapy. On the basis the type, the market has been segregated into mitochondrial, encephalomyopathy, lactic acidosis, and stroke-like episodes, progressive external ophthalmoplegia, myoclonic epilepsy with ragged red fibres, mitochondrial neurogastrointestinal encephalopathy syndrome, Kearns–Sayre syndrome, mitochondrial DNA depletion syndrome, Pearson syndrome, Leigh syndrome, neuropathy, ataxia, and retinitis pigmentosa. On the basis of diagnostic tests, the market has been bifurcated into genetic tests, muscle biopsy, biochemical test. Based on therapy, the market has been divided into supportive therapy and targeted therapy.


Regional Analysis


The global market for mitochondrial myopathy diagnosis & treatment has been segmented on the basis of regions into the Americas, Europe, Asia-Pacific, and Middle East & Africa. Among these has captured the whole market of mitochondrial myopathy at present as there are no targeted therapies available in the market. There are few molecules under clinical development for the targeted treatment of the mitochondrial myopathy, which is expected to be available in the market after 2021. However, supportive therapy will continue to dominate the global market until the availability of concrete treatment for the disease.


Americas mitochondrial myopathy market is the largest market for the diagnosis and treatment of mitochondrial myopathy. Advanced healthcare services, state of the art healthcare infrastructure, availability of the better healthcare services and outcomes, development in the genetic testing spectrum, high awareness about the mitochondrial diseases among the population, efforts taken by various organization for spreading the awareness about such rare disease conditions. All these factors culminate to make the American region the largest market in the world. Some of the companies such as GeneDx and ARUP laboratories, among others are involved in the genetic testing of the mitochondrial diseases, including myopathy. Moreover, few companies such as Stealth Biotherapeutics are engaged in the development of the novel drugs for the targeted therapies for the mitochondrial myopathies.



  • July 2017 Centogene AG signed a cooperation agreement with University Hospital St. Ivan Rilski in Bulgaria. The collaboration helped the company in expanding its genetic testing portfolio.



  • June 2017 Centogene AG raised nearly Euro 25 million in the financial round led by the TVM Capital Life Science in cooperation with DPE Deutsche Private Equity, Careventures, and CIC Capital. The financial boost enabled the company to provide a more extensive genetic testing portfolio to a larger customer base.


Market Segmentation and Key Market Players


Global Mitochondrial Myopathy Diagnosis & Treatment, by Type



  • Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes

  • Progressive External Ophthalmoplegia

  • Myoclonic Epilepsy with Ragged Red Fibers

  • Mitochondrial Neurogastrointestinal Encephalopathy Syndrome

  • Kearns–Sayre Syndrome

  • Mitochondrial DNA Depletion Syndrome

  • Pearson Syndrome

  • Leigh Syndrome

  • Neuropathy, Ataxia, and Retinitis Pigmentosa


Global Mitochondrial Myopathy Diagnosis & Treatment, by Diagnostic Test



  • Genetic Tests

  • Muscle Biopsy

  • Biochemical Test


Global Mitochondrial Myopathy Diagnosis & Treatment, by Therapy



  • Supportive Therapy

  • Targeted Therapy


Global Mitochondrial Myopathy Diagnosis & Treatment, by Key Players



  • AbbVie Inc.

  • Centogene AG

  • GeneDx

  • Ixchel Pharma

  • Khondrion BV

  • Mitobridge

  • NeuroVive Pharmaceutical AB

  • Reata Pharmaceuticals Inc.

  • Stealth Biotherapeutics


Intended Audience



  • Biotechnology companies

  • Hospitals

  • Research and development (R&D) companies

  • Market research and consulting service providers

  • Potential investors



Report Scope:
Report Attribute/Metric Details
  Market Size   2025: Significant Value
  CAGR   9.8% (2017-2025)
  Base Year   2019
  Forecast Period   2027
  Historical Data   2018
  Forecast Units   Value (USD Million)
  Report Coverage   Revenue Forecast, Competitive Landscape, Growth Factors, and Trends
  Segments Covered   Type, Diagnostic Test, Therapy
  Geographies Covered   North America, Europe, Asia-Pacific, and Rest of the World (RoW)
  Key Vendors   Stealth Biotherapeutics, Khondrion BV, NeuroVive Pharmaceutical AB, Reata Pharmaceuticals Inc., GeneDx, Mitobridge, Centogene AG, and Ixchel Pharma.
  Key Market Opportunities   New product launches and R&D Amongst major key Players
  Key Market Drivers   Rising prevalence and higher awareness regarding the disease and advancements


Frequently Asked Questions (FAQ) :


Mitochondrial myopathy diagnosis & treatment market projected to grow at approximately 9.8% CAGR during the assessment period (2020-2027).

The valuation of the global mitochondrial myopathy diagnosis & treatment market is estimated to increase to USD 33.6 MN by the end of 2025.

Rising prevalence and higher awareness regarding the disease and advancements in diagnosis & treatment process are major tailwinds pushing the growth of the global mitochondrial myopathy diagnosis & treatment market.

North America holds the largest share in the global mitochondrial myopathy diagnosis & treatment market, followed by Europe and the Asia Pacific, respectively.

Stealth Biotherapeutics, AbbVie Inc., Khondrion BV, Centogene AG, NeuroVive Pharmaceutical AB, Ixchel Pharma, Reata Pharmaceuticals Inc., Khondrion BV, GeneDx, Mitobridge, and Centogene AG, are some of the top players operating in the global mitochondrial myopathy diagnosis & treatment market.

1 Introduction

1.1 Definition 9

1.2 Scope of Study 9

1.3 Research Objective 9

1.4 Assumptions & Limitations 10

1.4.1 Assumptions 10

1.4.2 Limitations 10

1.5 Market Structure 11

2 Research Methodology

2.1 Research Process 12

2.2 Primary Research 13

2.3 Secondary Research 13

3 Market Dynamics

3.1 Introduction 14

3.2 Drivers 15

3.2.1 Rise in Number of Mitochondrial Myopathy Incidence Rate and Growing Number of Screening Services. 15

3.2.2 Government Funding and Incentives for Rare Genetic Diseases 15

3.3 Restraints 16

3.3.1 Poor Treatment Options and Absence of Curative Treatment 16

3.3.2 Lack of Awareness and Diagnosis of the Rare Mitochondrial Disorders 16

3.4 Opportunities 16

3.4.1 Faster Market Uptake and Low Marketing Cost 16

3.4.2 Research and Development 17

3.5 Challenges 17

3.5.1 Rare Nature of the Disease 17

3.5.2 Poor Understanding of Mitochondria and Genetics 17

3.5.3 Cost of Drug Development and High Investment Risk 17

4 Market Factor Analysis

4.1 Porter’s Five Forces Analysis 19

4.1.1 Threat from a New Entrant 19

4.1.2 Bargaining Power of Buyer 20

4.1.3 Bargaining Power of Supplier 21

4.1.4 Threat from Substitute 21

4.1.5 Intensity of Competitive Rivalry 21

4.2 Pipeline Analysis 23

4.2.1 Introduction 23

4.2.2 KH176 23

4.2.3 Elamipretide 24

4.2.4 RTA 408 25

4.2.5 IXC 103 26

4.2.6 NV556 26

4.3 Mega Trends 27

4.3.1 Strategies for Increasing Mitochondrial Biogenesis 27

4.3.2 Antioxidant Approaches 27

4.3.3 Targeting Mitochondrial Dynamics and Biogenesis 28

4.3.4 Mitochondrial Membrane Lipids as Therapeutic Targets 28

4.3.5 Nucleoside Replacement and Gene Therapy 28

4.3.6 Enzyme Replacement Therapy 28

4.4 Investment Opportunity Analysis 29

4.4.1 Market Size (Current and Future), Growth Rate, and Profitability 29

4.4.2 Investment Risks 29

4.4.3 Market Outlook 29

4.4.4 Competitors 30

4.4.5 Projections 30

5 Mitochondrial Myopathy Diagnosis & Treatment Market, By Type

5.1 Introduction 31

5.2 Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes (MELAS) 31

5.3 Progressive External Ophthalmoplegia (PEO) 31

5.4 Myoclonic Epilepsy with Ragged Red Fibres (MERRF) 32

5.5 Mitochondrial Neurogastrointestinal Encephalopathy Syndrome (MNGIE) 32

5.6 Kearns–Sayre Syndrome (KSS) 32

5.7 Mitochondrial DNA Depletion Syndrome (MDS) 32

5.8 Pearson Syndrome 33

5.9 Leigh Syndrome 33

5.10 Neuropathy, Ataxia, and Retinitis Pigmentosa (NARP) 33

6 Mitochondrial Myopathy Diagnosis & Treatment Market, By Diagnostic Test

6.1 Introduction 36

6.2 Genetic Tests 36

6.3 Muscle Biopsy 36

6.4 Biochemical Test 37

7 Mitochondrial Myopathy Diagnosis & Treatment Market, By Therapy

7.1 Introduction 39

7.2 Supportive Therapy 39

7.3 Targeted Therapy 40

8 Global Mitochondrial Myopathy Diagnosis & Treatment Market, By Region

8.1 Introduction 42

8.2 Americas 44

Mitochondrial Myopathy Diagnosis & Treatment Market, By Type

Mitochondrial Myopathy Diagnosis & Treatment Market, By Diagnostic Test

Mitochondrial Myopathy Diagnosis & Treatment Market, By Therapy

8.3 Europe 45

Mitochondrial Myopathy Diagnosis & Treatment Market, By Type

Mitochondrial Myopathy Diagnosis & Treatment Market, By Diagnostic Test

Mitochondrial Myopathy Diagnosis & Treatment Market, By Therapy

8.4 Asia-Pacific 47

Mitochondrial Myopathy Diagnosis & Treatment Market, By Type

Mitochondrial Myopathy Diagnosis & Treatment Market, By Diagnostic Test

Mitochondrial Myopathy Diagnosis & Treatment Market, By Therapy

8.5 Middle East & Africa 48

Mitochondrial Myopathy Diagnosis & Treatment Market, By Type

Mitochondrial Myopathy Diagnosis & Treatment Market, By Diagnostic Test

Mitochondrial Myopathy Diagnosis & Treatment Market, By Therapy

9 Company Profile

9.1 AbbVie Inc. 49

9.1.1 Company Overview 49

9.1.2 Product Overview 49

9.1.3 Financial Overview 49

9.2 Centogene AG 51

9.2.1 Company Overview 51

9.2.2 Product Overview 51

9.2.3 Financial Overview 51

9.2.4 Key Development 51

9.3 GeneDx 52

9.3.1 Company Overview 52

9.3.2 Product Overview 52

9.3.3 Financial Overview 52

9.3.4 Key Development 52

9.4 Ixchel Pharma 53

9.4.1 Company Overview 53

9.4.2 Product Overview 53

9.4.3 Financial Overview 53

9.5 Khondrion BV 54

9.5.1 Company Overview 54

9.5.2 Product Overview 54

9.5.3 Financial Overview 54

9.5.4 Key Development 54

9.6 Mitobridge 55

9.6.1 Company Overview 55

9.6.2 Product Overview 55

9.6.3 Financial Overview 55

9.7 NeuroVive Pharmaceutical AB 56

9.7.1 Company Overview 56

9.7.2 Product Overview 56

9.7.3 Financial Overview 56

9.7.4 Key Development 57

9.8 Reata Pharmaceuticals Inc. 58

9.8.1 Company Overview 58

9.8.2 Product Overview 58

9.8.3 Financial Overview 58

9.9 Stealth Biotherapeutics 60

9.9.1 Company Overview 60

9.9.2 Product Overview 60

9.9.3 Financial Overview 60

9.9.4 Key Developments 60

10 List of Tables

TABLE 1 MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET: PIPELINE ANALYSIS 23

TABLE 2 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY TYPE, 2020 –2027 (USD MILLION) 34

TABLE 3 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY TYPE 2021 – 2027 (USD MILLION) 35

TABLE 4 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY DIAGNOSTIC TEST, 2020–2027 (USD MILLION) 38

TABLE 5 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY DIAGNOSTIC TEST, 2021–2027 (USD MILLION) 38

TABLE 6 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY THERAPY, 2020–2027 (USD MILLION) 40

TABLE 7 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY THERAPY, 2021–2027 (USD MILLION) 41

TABLE 8 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY REGION, 2020–2027 (USD MILLION) 43

TABLE 9 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET, BY REGION, 2021–2027 (USD MILLION) 44

TABLE 10 AMERICAS: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2020–2027 (USD MILLION) 44

TABLE 11 AMERICAS: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2021–2027 (USD MILLION) 45

TABLE 12 EUROPE: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2020–2027 (USD MILLION) 45

TABLE 13 EUROPE: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2021–2027 (USD MILLION) 46

TABLE 14 ASIA-PACIFIC: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2020–2027 (USD MILLION) 47

TABLE 15 ASIA-PACIFIC: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2021–2027 (USD MILLION) 47

TABLE 16 MIDDLE EAST & AFRICA: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2020–2027 (USD MILLION) 48

TABLE 17 MIDDLE EAST & AFRICA: MITOCHONDRIAL MYOPATHIES DIAGNOSIS & TREATMENT MARKET, BY REGION, 2021–2027 (USD MILLION) 48

11 List of Figures

FIGURE 1 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET: MARKET STRUCTURE 11

FIGURE 2 RESEARCH PROCESS OF MRFR 12

FIGURE 3 FORECAST INDICATORS 14

FIGURE 4 PORTERS FIVE FORCES MODEL 19

FIGURE 5 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET BY TYPE, 2016, 2021 & 2027 (USD MILLION) 34

FIGURE 6 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET BY DIAGNOSTIC TEST, 2016, 2021 & 2027 (USD MILLION) 37

FIGURE 7 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET BY THERAPY, 2016, 2021 & 2027 (USD MILLION) 40

FIGURE 8 GLOBAL MITOCHONDRIAL MYOPATHY DIAGNOSIS & TREATMENT MARKET BY REGION, 2016, 2021 & 2027 (USD MILLION) 43

FIGURE 9 ABBVIE: RECENT FINANCIAL 49

FIGURE 10 ABBVIE: GEOGRAPHIC MIX, 2016 (%) 50

FIGURE 11 NEUROVIVE PHARMACEUTICAL AB: RECENT FINANCIALS 56

FIGURE 12 REATA PHARMACEUTICALS: RECENT FINANCIALS 58

FIGURE 13 REATA PHARMACEUTICALS: R&D EXPENSES, 2020 (%) 59