New York, USA, July 24, 2024 (GLOBE NEWSWIRE) -- Leigh Syndrome Market Anticipates Impressive Growth Trajectory Across 7MM During the Study Period (2020–2034) | DelveInsight
DelveInsight’s analysts estimate that the Leigh syndrome market is expected to show positive growth, mainly attributed to increased prevalence and the anticipated launch of novel therapies during the forecast period (2024–2034).
DelveInsight’s Leigh Syndrome Market Insights report includes a comprehensive understanding of current treatment practices, Leigh syndrome emerging drugs, market share of individual therapies, and current and forecasted Leigh syndrome market size from 2020 to 2034, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].
Key Takeaways from the Leigh Syndrome Market Report
- According to DelveInsight’s analysis, the market size of Leigh syndrome in the 7MM is expected to grow at a significant CAGR by 2034.
- The prevalence reported for Leigh syndrome is 1 in 30,000 to 1 in 40,000 people at birth. Mitochondrial DNA-associated Leigh syndrome, which is rarer than nuclear gene-encoded Leigh syndrome, is likely to occur in about 1 in 100,000 to 1 in 140,000 births.
- Prominent companies working in the domain of Leigh syndrome, including PTC Therapeutics, Abliva AB, and others, are actively working on innovative drugs for Leigh syndrome. These novel Leigh syndrome therapies are anticipated to enter the Leigh syndrome market in the forecast period and are expected to change the market.
- Some of the key therapies for Leigh syndrome treatment include Vatiquinone, NV354, and others.
Discover which therapies are expected to grab Leigh syndrome market share @ Leigh Syndrome Market Report
Leigh Syndrome Overview
Leigh syndrome is a rare genetic disorder affecting neurometabolism. It is marked by the deterioration of the central nervous system, including the brain, spinal cord, and optic nerve. Typically, Leigh syndrome is passed down as an autosomal recessive trait, but it can also be inherited through X-linked recessive or maternal transmission due to mitochondrial DNA mutations.
The symptoms of Leigh syndrome often worsen quickly. Initial signs may include difficulty sucking, loss of head control and motor skills, decreased appetite, vomiting, and seizures. As the disease advances, it may cause muscle weakness, reduced muscle tone, spasticity, movement disorders, cerebellar ataxia, and peripheral neuropathy. Complications can affect respiratory, heart, and kidney functions.
Diagnosing Leigh syndrome involves a detailed clinical evaluation and various specialized tests, particularly advanced imaging methods. MRI or CT scans of the brain can reveal abnormalities in specific areas such as the basal ganglia, brain stem, and gray matter.
Leigh Syndrome Epidemiology Segmentation
The Leigh syndrome epidemiology section provides insights into the historical and current Leigh syndrome patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The Leigh syndrome market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
- Total Prevalent Cases of Leigh Syndrome
- Onset-specific Prevalent Cases of Leigh Syndrome
- Mutation-specific Prevalent Cases of Leigh Syndrome
- Gender-specific Prevalent Cases of Leigh Syndrome
- Diagnosed and Treatable Cases of Leigh Syndrome
Download the report to understand which factors are driving Leigh syndrome epidemiology trends @ Leigh Syndrome Epidemiological Insights
Leigh Syndrome Treatment Market
There is no particular treatment for Leigh syndrome, and generally, specific therapies for mitochondrial disorders in children are not widely accessible. Symptomatic treatment aims to manage symptoms and enhance energy levels by boosting ATP production and reducing lactate levels. This is achieved through the elimination of harmful metabolites and the use of oxygen radical scavengers. Antioxidants are frequently used in treatment. Symptomatic treatments for Leigh syndrome include supplementation with B vitamins like thiamine, antiseizure medications, physiotherapy, and pharmacotherapy.
Various substances, commonly known as 'nutraceuticals' because they are derived from everyday foods, have been suggested for treating LS or other mitochondrial disorders. These substances include coenzyme Q10, L-carnitine, alpha-lipoic acid, creatine monohydrate, biotin, thiamine, riboflavin, among others. Often, patients receive a combination of these compounds as part of their treatment.
Symptomatic treatments, such as antiseizure medication to manage seizures, are crucial components of care. Controlling seizures without additional side effects can greatly enhance quality of life. Addressing neuromuscular issues through physiotherapy and medication, using proper feeding techniques to prevent malnutrition and aspiration, and promptly treating fever and infections are also key aspects of treatment aimed at improving quality of life.
Learn more about the FDA-approved drugs for Leigh syndrome @ Drugs for Leigh Syndrome Treatment
Leigh Syndrome Emerging Drugs and Companies
The market has a promising outlook with some of the potential emerging therapies for the treatment of Leigh syndrome. Some of the drugs in the pipeline include Vatiquinone (PTC Therapeutics), and NV354 (Abliva AB), among others.
Vatiquinone, derived from alpha tocotrienol, a form of vitamin E, is the resulting compound after oxidation. It acts on the enzyme 15-lipoxygenase, crucial in managing oxidative stress and inflammation across various tissues. By inhibiting this enzyme, it reduces inflammation and oxidative stress, thereby enhancing cellular health.
In October 2019, the company obtained the assets of BioElectron, which included EPI-743. Back in June 2014, the FDA granted orphan drug designation for Vatiquinone for treating Leigh Syndrome. Presently, Vatiquinone is undergoing Phase III trials to evaluate its safety in individuals with inherited mitochondrial disorders who were previously exposed to it in earlier clinical studies or treatment plans sponsored by PTC/BioElectron. This trial encompasses patients with Leigh syndrome as well.
NV354 is in the developmental stages for treating mitochondrial disease, particularly targeting insufficient activity in mitochondrial protein complex I, which leads to a deficiency in energy conversion and manifests as various neurologic complications in diseases like Leigh syndrome, MELAS, or LHON. This drug candidate was identified for its ability to enhance mitochondrial function in cells of patients with mitochondrial Leigh syndrome.
NV354, which can penetrate the brain, is a product of innovation wherein the body's own energy substrate, succinate, is made available in cells using prodrug technology. Preclinical development for this program has been finalized, and in April 2023, the drug received orphan drug designation (ODD) in the US for mitochondrial disease treatment. Additionally, in December 2023, Abliva secured orphan designation for NV354 from the European Commission specifically for treating Leigh syndrome.
The anticipated launch of these emerging therapies are poised to transform the Leigh syndrome market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the Leigh syndrome market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth.
To know more about Leigh syndrome clinical trials, visit @ Leigh Syndrome Treatment Drugs
Leigh Syndrome Market Dynamics
The Leigh syndrome market dynamics are anticipated to change in the coming years. The Leigh syndrome market is primarily driven by increased awareness and diagnosis of the condition, advancements in genetic testing, and a growing focus on rare disease research. Enhanced funding from both governmental and non-governmental organizations for mitochondrial disorder studies has accelerated the development of novel therapeutic approaches. Additionally, the rise of patient advocacy groups has facilitated greater collaboration among researchers, healthcare providers, and pharmaceutical companies, fostering a more supportive environment for the development and approval of new treatments. The expanding pipeline of potential therapies and the ongoing efforts to improve clinical trial methodologies further contribute to the market's growth potential.
Furthermore, many potential therapies are being investigated for the treatment of Leigh syndrome, and it is safe to predict that the treatment space will significantly impact the Leigh syndrome market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the Leigh syndrome market in the 7MM.
However, several factors may impede the growth of the Leigh syndrome market. The complexity of the syndrome, stemming from its genetic heterogeneity and variable clinical presentations, further complicates the development of effective therapies. Additionally, there are substantial challenges related to diagnosis and patient identification, as well as a lack of comprehensive natural history studies that can inform clinical trial design. Regulatory hurdles and the high cost of drug development for rare diseases also pose significant obstacles, making it difficult for new treatments to reach the market and patients who desperately need them.
Moreover, Leigh syndrome treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, Leigh syndrome market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact Leigh syndrome market growth.
| Leigh Syndrome Report Metrics | Details |
| Study Period | 2020–2034 |
| Leigh Syndrome Report Coverage | 7MM [The United States, the EU-4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] |
| Key Leigh Syndrome Companies | PTC Therapeutics, Abliva AB, and others |
| Key Leigh Syndrome Therapies | Vatiquinone, NV354, and others |
Scope of the Leigh Syndrome Market Report
- Leigh Syndrome Therapeutic Assessment: Leigh Syndrome current marketed and emerging therapies
- Leigh Syndrome Market Dynamics: Attribute Analysis of Emerging Leigh Syndrome Drugs
- Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Leigh Syndrome Market Access and Reimbursement
Discover more about Leigh syndrome drugs in development @ Leigh Syndrome Clinical Trials
Table of Contents
| 1. | Leigh Syndrome Market Key Insights |
| 2. | Leigh Syndrome Market Report Introduction |
| 3. | Leigh Syndrome Market Overview at a Glance |
| 4. | Leigh Syndrome Market Executive Summary |
| 5. | Disease Background and Overview |
| 6. | Leigh Syndrome Treatment and Management |
| 7. | Leigh Syndrome Epidemiology and Patient Population |
| 8. | Patient Journey |
| 9. | Leigh Syndrome Marketed Drugs |
| 10. | Leigh Syndrome Emerging Drugs |
| 11. | Seven Major Leigh Syndrome Market Analysis |
| 12. | Leigh Syndrome Market Outlook |
| 13. | Potential of Current and Emerging Therapies |
| 14. | KOL Views |
| 15. | Unmet Needs |
| 16. | SWOT Analysis |
| 17. | Appendix |
| 18. | DelveInsight Capabilities |
| 19. | Disclaimer |
| 20. | About DelveInsight |
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