Number of pages: 100 | Report Format: PDF | Published date: February 14, 2023
Historical Years – 2020 | Base Year – | Forecasted Years – 2022-2030
According to the deep-dive market assessment study by Growth Plus Reports, the global myotonic dystrophy treatment market was valued at US$ 735.2 million in 2021 and is expected to register a revenue CAGR of 5.56% to reach US$ 1,200 million by 2030.
Myotonic Dystrophy Treatment Market Fundamentals
Myotonic dystrophy is a complex multisystem disorder affecting many body organs. It is the most frequent kind of adult-onset. The condition is characterized by gradual muscular atrophy and weakening. Symptoms of myotonic dystrophy mostly begin in childhood or the adult years. Most individuals experience heavy muscle cramps, scoliosis, vision problems, and trouble running and walking.
There are two main types of myotonic dystrophy (DM), DM1 and DM2, both of which are brought on by chromosomal abnormalities. This condition causes multisystem problems, such as skeletal muscle weakness and myotonia (difficulty relaxing muscles), heart irregularities, cataracts, and other complications.
The most prevalent form, DM1 myotonia dystrophy, is caused by an aberrant DNA expansion in the DMPK gene on chromosome 19. The ZNF9 gene on chromosome 3 is the source of an aberrant DNA expansion that leads to DM2. In myotonic dystrophy type 1, the repetitive expansion grows with each aggrieved generation, typically causing an earlier start and worsening symptoms.
DM2, also known as PROMM (proximal myotonic myopathy), does not have a congenital start and rarely develops in childhood. DM2 is more likely to affect the proximal muscles and distal muscles, which are the first to be affected in DM1. In comparison to typical DM1, DM2 often has less severe symptoms. However, as it involves the early weakening of the hip muscles, it may impair walking ability earlier than DM1.
Myotonic Dystrophy Treatment Market Dynamics
According to Myotonic Dystrophy Foundation, myotonic dystrophy is a rare, multi-systemic genetic condition that impacts 1 in 2,100 individuals or more than 3.6 million people globally. DM 1 is the most common myotonic dystrophy type. According to the American Academy of Neurology, there are currently between 5 to 20 cases of DM1 per 100,000 people.
The increasing prevalence of myotonic dystrophy is raising the demand for new and innovative drugs in the market. Hence, top players are heavily investing in launching new drugs in the market. For instance, in December 2022, Arthrex Biotech S.A., a pre-clinical-stage biotechnology firm pioneering unique microRNA modifying approaches to diseases with significant unmet medical needs, reached major regulatory milestones in its program to develop ATX-01 in Myotonic Dystrophy Type 1 (DM1), clearing the way for the submission of an Investigational New Drug (IND) application in the United States and a Clinical Trial Application (CTA) in Europe.
The global myotonic dystrophy treatment market is expected to experience high revenue growth during the forecast period, owing to increase in the pipeline drugs being studied in clinical trials. For instance, AM0-02, or Tideglusib, is a clinical-stage experimental drug for the treatment of DM1. The activity of glycogen synthase kinase 3 beta (GSK3ß) has been reported to rise in DM1 cellular and animal models, as well as in muscle biopsies from patients. AMO-02 is an inhibitor that has been demonstrated to normalize GSK3ß levels in transgenic models and ex vivo tissue samples from DM1 patients, as well as to diminish levels of mRNA that is harmful to DM1.
However, the high cost of drugs due to the high expenditures associated with their manufacturing is likely to restrain market revenue growth potential to some extent.
Myotonic Dystrophy Treatment Market Ecosystem
The global myotonic dystrophy treatment market has been analyzed from three perspectives: therapeutic class, distribution channel, and region.
Myotonic dystrophy treatment market by Therapeutic Class
Based on therapeutic class, the global myotonic dystrophy treatment market is segmented into molecular-based therapies and steroid therapy.
The molecular-based therapy segment accounted for the highest market revenue share in 2021. Molecular-based therapy in the treatment of myotonic dystrophy is intended to target the mutant allele, its RNA product, or its downstream signaling pathways specifically. The molecular-based therapy segment is sub-segmented into DMPK gene expression and CUG transcript. The DMPK gene expression is used to increase the expression of the DMPK gene. It reduces muscle wasting, improves muscle strength control, and improves balance and coordination. Additionally, it has been found that gene expression in the DMPK gene can improve cognitive function, reduce fatigue, and improve the overall quality of life. Thus, gene expression in the DMPK gene can be a powerful tool in treating myotonic dystrophy and improving the quality of life for those affected by this disorder.
The steroid segment is expected to show lucrative revenue growth during the forecast period. This is attributed to the launch of new steroids in the market. Moreover, healthcare professionals prescribe steroids for short-term use, especially when respiration and swallowing are impaired. Steroids, according to experts, assist in reducing inflammation and impact the regeneration of compromised muscle cell membranes. The steroid segment is sub-segmented into prednisone and deflazacort. Prednisone and deflazacort improve muscular strength and function in people with myotonic dystrophy.
Myotonic dystrophy treatment market by Distribution Channel
Based on the distribution channel, the global myotonic dystrophy treatment market is segmented into hospital pharmacy, retail pharmacy, and online pharmacy.
The hospital pharmacy segment accounted majority of the market revenue share in 2021. The rise in healthcare costs has caused many patients to seek more medications, which has fueled the revenue growth of the hospital pharmacy segment. Additionally, quality assurance and safety standards of hospital pharmacies provide an additional benefit to the patients. Furthermore, the segment’s growth is also supported by high patient footfall in hospitals.
Myotonic dystrophy treatment market by Region
Regionally, the global myotonic dystrophy treatment market has been segmented into North America, Europe, Asia Pacific, and the rest of the world.
North America dominated the market with the highest market revenue share in 2021. The myotonic dystrophy treatment market growth in North America is attributed to the rising prevalence of myotonic dystrophy in the region.
The market in Europe is characterized by an increasing prevalence of type 2 myotonic dystrophy. In Germany and Finland, myotonic dystrophy type 2 is more prevalent than myotonic dystrophy type 1, accounting for about 5,000 cases in 2019.
The Asia Pacific market is anticipated to register high revenue growth during the forecast period due to increasing healthcare spending by governments and private organizations, as well as growing awareness about myotonic dystrophy disease treatments.
The market in the middle east and Africa is anticipated to experience significant growth during the forecast period as a result of an increased focus on drug development programs by various pharmaceutical companies, particularly those from Israel, which are aimed at addressing diseases common to this region.
Myotonic Dystrophy Treatment Market Competitive Landscape
The prominent players operating in the global myotonic dystrophy treatment market are:
The global myotonic dystrophy treatment market is being driven by the increasing prevalence of the disorder globally. Furthermore, increasing research and development activities to launch new drugs and growing efforts of many private and government organizations to spread awareness about the disease are fueling revenue growth in the global market.
Lupin Ltd., Pfizer, Inc, Eli Lilly and Company, Novartis AG, and Teva Pharmaceutical Industries Ltd. are among the top market players.
The high cost of the drugs due to the high expenditures associated with their manufacturing is one of the major factors which can limit the market potential.
The global myotonic dystrophy treatment market is expected to register a revenue CAGR of 5.56 % during the forecast period from 2022 to 2030.
The molecular-based therapy accounted for the majority of the market revenue share in 2021.
*Insights on financial performance are subject to the availability of information in the public domain.