The global Nucleic Acid And Gene Therapies In Neuromuscular Disorders market size is expected to be valued at USD 7.88 Billion in 2023. With the increasingly high incidence of cancer and other target diseases, the overall demand for Nucleic Acid And Gene Therapies In Neuromuscular Disorders is projected to grow at a CAGR of 9.5% between 2023 and 2033, totaling around USD 19.52 Billion by 2033.
| Data Points | Key Statistics |
|---|---|
| Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market Value 2023 | USD 7.88 Billion |
| Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market Projected Value (2033) | USD 19.52 Billion |
| Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market CAGR (2023 to 2033) | 9.5% |
According to the analysis of Future Market Insights, the Nucleic Acid And Gene Therapies In the Neuromuscular Disorders market are expected to fuel over the analysis period, attributed to the factors such as a rise in the number of drug approvals, which will lead to the expansion of the market growth.
Moreover, owing to the rapidly progressive nature of the disease, the treatments using drugs are not effective and do not stop the disease progression. This leads the vendors to conduct research on the development of gene therapies and antisense therapies that can stop the progression of the disease and increase the life expectancy of the patients.
An upsurge in technological advances is a major factor that is projected to contribute significantly to the research and development of novel therapeutics for neuromuscular diseases. In addition, the lack of efficacy and non-disease-modifying drugs for the treatment are leading the vendors to focus on identifying the benefits of regenerative therapies as a potential treatment option.
Furthermore, the advent of next-generation genetic sequencing technology focuses on sequencing the human genome, while significantly reducing the associated costs. This, in turn, results in the market witnessing advances in the identification of mutated genes that cause neuromuscular diseases. This development is expected to have a positive impact on overall market growth.
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The global demand for Nucleic Acid And Gene Therapies In Neuromuscular Disorders is projected to increase at a CAGR of 9.5% during the forecast period between 2023 and 2033, reaching a total of USD 19.52 Billion in 2033, according to a report from Future Market Insights (FMI). From 2018 to 2022, sales witnessed significant growth, registering a CAGR of 3.5%.
According to Future Market Insights, a market research and competitive intelligence provider, the Nucleic Acid And Gene Therapies In the Neuromuscular Disorders market were valued at USD 7.19 Billion in 2022.
Factors including a surge in government initiatives to ensure early diagnosis, an increase in awareness about genetic disorders, enhanced healthcare infrastructure, and a rise in research and development activities are anticipated to propel the growth of nucleic acid and gene therapies in the neuromuscular disorders market during the forecast period.
According to a study conducted in 2014, the prevalence rate of neuromuscular disorders was between 1 and 10 per 100,000, except for multifocal motor neuropathy, Lambert-Eaton myasthenic syndrome, Emery-Dreifuss dystrophy, oculopharyngeal muscular dystrophy, and congenital muscular dystrophies, which was <1/100,000. Post-polio syndrome and Charcot-Marie-Tooth disease had a prevalence of >10/100,000.
Diagnosis of neuromuscular disorders is performed through physical examination as well as certain diagnostic tests. A physician runs a complete medical history assessment, in order to assess the condition of a patient to determine if any family member had previously had symptoms of neuromuscular disorders. Currently, there is no cure for neuromuscular disorders. However, research is being done on genetic therapies and new medications to develop a cure. These are lifelong disorders and cannot be cured completely, but can be managed to help individuals to maximize their capabilities.
Rise in R&D Activities to Fuel the Market Growth
Neuromuscular diseases are a broad category of disorders that cause progressive motor function impairment. Motor neurons, skeletal muscle, or their synaptic connection, the neuromuscular junction, are the most common Neuromuscular Disorders. MNs [e.g., amyotrophic lateral sclerosis caused by direct loss or retrograde degeneration of MNs], muscle [e.g., Duchenne muscular dystrophy or myotonic dystrophy], NMJs [e.g., myasthenia gravis or congenital myasthenic syndromes], or a combination of these [e.g. (e.g., Pompe disease).
Moreover, regardless of its source, the structural and functional deficit in a targeted tissue will resonate throughout the motor unit, resulting in multiple shared symptoms among different Neuromuscular Disorders.
Methods to differentiate hiPSCs into neuronal cells have advanced rapidly in the last decade, because of an increased understanding of early neural development and the commitment of neuro progenitor cells to highly specialized neural subtypes, including MNs. The MNs are found throughout the CNS and are divided into upper MNs (UMNs) and lower MNs (LMNs), which are developmentally and genetically distinct despite their shared nomenclature. Different NMDs can target UMNs and LMNs, making their distinction important when modeling NMDs. The cerebral cortex's pre-motor and primary motor regions are where UMNs originate. As a result, many recent breakthroughs in neuromuscular disease therapy development have been made.
There are approximately 200 products in the pipeline for neuromuscular disorders, with a roughly 50:50 ratio of preclinical to clinical development. Most target amyotrophic lateral sclerosis (ALS) and Duchenne's muscular dystrophy (DMD). Recent technological advancements have aided the development of several different types of therapies. In 2018, small molecules (targeting receptor modulation, epigenetic reprogramming, redox metabolism, and other mechanisms), gene therapy, and antisense oligonucleotides accounted for nearly half of the nearly 200 potential therapies for neuromuscular diseases under development (Aitken et al., 2018). The US Food and Drug Administration recently approved ASO and gene therapy. Thus, these factors are anticipated to fuel market growth in the forthcoming years.
Novel Drug Approvals to Accelerate the Market Growth
Novel drug approvals are one of the key factors that are projected to augment the growth of nucleic acid and gene therapies in the neuromuscular disorders market. There has been an increase in drug approvals in the market for treating various neuromuscular indications in recent years. This is attributed to the advanced research being conducted on highly morbid and progressive indications.
Spinal muscular atrophy is one of the major neuromuscular diseases for which the market has witnessed therapeutic approvals. Spinal muscular atrophy is morbid and rapidly progressive neuromuscular. It is caused by the death of a type of nerve cell, called motor neurons, in the spinal cord. Treatments using drugs are not effective and do not stop the disease progression due to the rapidly progressive nature of the disease.
To overcome this challenge, various vendors are conducting research on the development of novel gene therapies and antisense therapies that can stop the progression of the disease and increase the life expectancy of patients. Hence, these factors are expected to accelerate the growth of the market growth during the forecast period.
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Exorbitant Treatment Costs to Restrain the Market Growth
Regardless of the increasing prevalence of chronic disorders such as SMA and cancer, the treatment method for neuromuscular disorders is extremely expensive. The method is majorly advertised as a single-dose treatment regime that reverses the genetic dysfunction in the patient’s body. As per Novartis AG, a single dose of Kymriah, the therapy cancer drug costs a whopping USD 475,000. The same drug is priced at USD 306,000 in Japan.
This expensive therapy has also encouraged reluctance to reimburse the treatment method by some insurance companies. This is one of the major factors that is expected to hinder the growth of nucleic acid and gene therapies in the neuromuscular disorders market during the forecast period.
Additionally, low awareness in developing countries and a lack of diagnostic facilities are other factors that will hamper market growth in the near future.
Side Effects Associated with Neuromuscular Therapeutics to Limit the Market Growth
Myasthenia gravis (MG) is a neuromuscular junction disorder that causes varying degrees of weakness in the body's skeletal muscles. Although there is currently no cure for MG, traditional therapies such as acetylcholinesterase inhibitors as well as immunomodulating therapies (corticosteroids, azathioprine, and mycophenolate mofetil) enable most patients to live productive lives with an average life expectancy.
However, these therapies are associated with side effects such as nausea, vomiting, gastrointestinal (GI) upset, increased infection risk, weight gain, and liver damage, which can deter patients from using the medications.
Additionally, poor adherence is also a result of unsatisfactory treatment outcomes with traditional MG therapies, with 23 percent of patients reporting poor treatment compliance. Despite this, patient-reported information on the exact nature, duration, and severity of these side effects is scarce. Moreover, stringent regulations imposed by governments are another major factor that is expected to impede market growth over the analysis period.
Better Healthcare Infrastructure in the Region to Fuel the Market Growth
The Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market in North America is expected to accumulate the highest market share of 46% in 2023.
Factors such as the increasing patient population, presence of major market players in North America, and better healthcare infrastructure are projected to boost the regional market growth during the forecast period. Moreover, increasing approvals of novel nucleic acid treatments and the availability of nucleic acid extraction technology are other factors that are expected to accelerate the market growth in the region. For instance, neuromuscular diseases (NMDs) are a broad category of disorders that affect an estimated 250,000 people in the United States.
In addition, in December 2021, Vyvgart (efgartigimod) received approval from the US Food and Drug Administration for the treatment of generalized myasthenia gravis (gMG) in adults who test positive for the anti-acetylcholine receptor (AChR) antibody. The region is expected to hold the highest CAGR of 9.4% during the forecast period.
Increasing Population in the Region to Fuel the Market Growth
The Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market in Asia Pacific are expected to accumulate the highest market share of 44% in 2023.
The Asia Pacific hypophosphatasia market is anticipated to register significant growth during the analysis period, attributed to factors such as an increase in population, high acceptance of new treatment methodologies, as well as improvement in healthcare infrastructure in countries such as India. Furthermore, the rise in focus of major market players in Asia Pacific is another major factor that will propel the revenue share of the regional market during the forecast period.
An increase in investment, strong collaboration among research institutes and private players, and high awareness regarding the treatment are other factors that are anticipated to escalate the growth of the market in the region in the near future. The region is expected to hold the highest CAGR of 9.3% during the forecast period.
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Spinal Muscular Atrophy Segment to Drive the Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market
Based on the Therapy, the Spinal Muscular Atrophy segment is expected to witness a significant growth of 46% in 2022, and the trend is expected to continue during the forecast period, expanding at a rapid rate of 9.4% CAGR over the analysis period.
The segment’s growth is attributed to the SMA being a good example of a monogenic disease that currently has an AAV9-based vector gene therapy as a therapeutic option. In addition, the rise in the incidence of cancer and other target diseases, the availability of reimbursement policies, as well as the increase in funding for gene therapy research are other major factors escalating the growth of the segment.
Hospitals Segment to Drive the Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market
Based on the Application, the hospital's segment is expected to witness a significant growth of 45% in 2022, and the trend is expected to continue during the forecast period, expanding at a rapid rate of 9.3% CAGR over the analysis period.
In terms of application, the hospital segment is expected to have the highest share of the market during the forecast period. The dominance is attributed to the high availability as well as the accessibility of the treatment methods in such facilities.
Nucleic Acid And Gene Therapies In Neuromuscular Disorders Market startup players are adopting various marketing strategies such as new product launches, geographical expansion, mergers and acquisitions, partnerships, and collaboration to identify the interest of potential patients and create a larger customer base. For instance,
Prominent players in the Nucleic Acid And Gene Therapies In Neuromuscular Disorders market are Pfizer, Inc., F. Hoffmann-La Roche Ltd., UCB Pharma, Biogen, Astellas Pharma, Inc., Novartis AG, Abbott Laboratories, Inc., and Sanofi, among others.
Recent Developments:
| Report Attribute | Details |
|---|---|
| Growth Rate | CAGR of 9.5% from 2023 to 2033 |
| Market Value in 2023 | USD 7.88 Billion |
| Market Value in 2033 | USD 19.52 Billion |
| Base Year for Estimation | 2022 |
| Historical Data | 2018 to 2022 |
| Forecast Period | 2023 to 2033 |
| Quantitative Units | Revenue in USD Billion and CAGR from 2023 to 2033 |
| Report Coverage | Revenue Forecast, Company Ranking, Competitive Landscape, Growth Factors, Trends, and Pricing Analysis |
| Segments Covered | Disorder, Therapy, Application, Region |
| Regions Covered | North America; Europe; Asia Pacific; Latin America; Middle East & Africa (MEA) |
| Key Countries Profiled | The US, Canada, Brazil, Mexico, Germany, The UK, France, Spain, Italy, China, Japan, South Korea, Malaysia, Singapore, Australia, New Zealand, GCC, South Africa, Israel |
| Key Companies Profiled | Pfizer, Inc.; F. Hoffmann-La Roche Ltd.; UCB Pharma; Biogen; Astellas Pharma, Inc.; Novartis AG; Abbott Laboratories, Inc.; Sanofi |
As of 2022, the market for Nucleic Acid And Gene Therapies In Neuromuscular Disorders was valued at USD 7.19 Billion, as per FMI
By 2023-end, sales of Nucleic Acid And Gene Therapies In Neuromuscular Disorders closed at a value of USD 7.88 Billion
From 2018 to 2022, Nucleic Acid And Gene Therapies In Neuromuscular Disorders demand expanded at a CAGR of 3.5%
From 2023 to 2033, Nucleic Acid And Gene Therapies In Neuromuscular Disorders sales are expected to flourish at a CAGR of 9.5%
By 2033, the market value of Nucleic Acid And Gene Therapies In Neuromuscular Disorders is expected to reach USD 19.52 Billion.
By Therapy, the Spinal Muscular Atrophy segment constitutes the bulk of market share of 46% in 2022.
By Application, the Hospitals segment dominates the market with a share of 45% in 2022.
1. Executive Summary | Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market 2. Market Overview 3. Market Background 4. Global Market Analysis 2018 to 2022 and Forecast, 2023 to 2033 5. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Disorder 5.1. Spinal Muscular Atrophy 5.2. Duchenne Muscular Dystrophy 5.3. Limb-girdle Muscular Dystrophy 5.4. Myotubular Myopathy 5.5. Hereditary Motor and Sensory Neuropathy Type 5.6. Amyloid Transthyretin 6. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Therapy 6.1. Splice-switching ASO 6.2. Gapmer ASO 6.3. Liposome-delivered siRNA 6.4. Delivery of SBillion 6.5. Gene Augmentation 7. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Application 7.1. Hospitals 7.2. Academic & Research Institutes 8. Global Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Region 8.1. North America 8.2. Latin America 8.3. Europe 8.4. Asia Pacific 8.5. Middle East & Africa (MEA) 9. North America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 10. Latin America Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 11. Europe Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 12. Asia Pacific Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 13. MEA Market Analysis 2018 to 2022 and Forecast 2023 to 2033, By Country 14. Key Countries Market Analysis 15. Market Structure Analysis 16. Competition Analysis 16.1. Celsion Corporation 16.2. Wave Life Sciences 16.3. Imugene 16.4. Caperna 16.5. PYC Therapeutics Ltd. 16.6. Oxford Biomedica 16.7. Benitec Biopharma Ltd 16.8. EdiGene, Inc. 16.9. GenScript 16.10. Arctoris Limited 17. Assumptions & Acronyms Used 18. Research Methodology
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Nucleic Acid and Gene Therapies in Neuromuscular Disorders Market
