Muscular dystrophy (MD) is a group of rare neuromuscular diseases that causes gradual weakening and loss of muscular mass. It is caused due to the interference of some abnormal genes with the production of proteins required for healthy muscles. There are over 30 different types of MD disorders that may be present at birth or appear during childhood or adulthood. Currently, there is no cure for this disease, but therapy and medications may help to control the symptoms. Various drugs like corticosteroids, creatine supplements, and heart medicines are prescribed for strengthening the muscles.
The muscular dystrophy drugs market is projected to witness strong growth in the upcoming years. This can majorly be attributed to the increase in various experiments and clinical trials conducted by the pharmaceutical industries. These trials focus on the development of potential treatments for MD. One of the most common types of MD, called Duchenne muscular dystrophy (DMD) can be treated with the help of exon-skipping therapy. Exons are part of a DNA that are involved in coding for proteins. This therapy prevents the mutation of the genes, by skipping over the exon responsible for the disease. Since it has shown favorable results, this therapy may also help to drive the market. As there is no permanent cure for MD, available treatments primarily focus on the management of symptoms. The development of more efficacious drugs for the treatment of MD, with fewer side effects, will fuel this market in the upcoming years. There is an unmet need for these drugs, driving the research and development organizations. There has been an increasing prevalence of MD in the last few years. Some factors like increased awareness among the people and better diagnostic methods may also act as a driver for this market.
The MD market may witness some challenges, restricting its growth. Most of the drugs used to treat MD have a high cost. This can act as a barrier for a large group of patients who cannot afford expensive medication. These drugs have limited efficacy that differs from patient to patient. Drugs may be effective in certain types of diseases only. This makes it challenging to find an effective cure for a particular type of MD. Also, there are various regulatory barriers to the development of drugs for rare diseases like this. Since it is a rare disease, a small population is affected by MD. Therefore, there is minimal investment in research and development for this drugs.
The market share for MD drugs is anticipated to witness several new opportunities in the forthcoming years. Novel treatment strategies like the advent of gene therapy techniques and stem cell therapy may provide significant breaks for the key players in the market. Many companies are now attempting to develop innovative strategies and improve the therapies already in use. Because of the rising incidence, the government is interested in funding various research and clinical trials as well as raising public awareness. This has caused an increase in the research and development programs for MD drugs. Owing to these factors, the market is expected to grow during the forecast period.
New product trends to flourish in the market
In August 2020 U.S. FDA approved, Zolgensma, the first gene therapy for the treatment of MD. In recent years, gene therapy has become a promising approach for MD. This treatment makes use of a virus to deliver a copy of a particular gene, namely the SMN1 gene to patients suffering from spinal muscular atrophy. Other than this, various combination therapies are under study for more favorable outcomes. For instance, the combination of exon-skipping therapy with gene therapy is under clinical trials.
Segment overview
By drugs: The MD market is classified as corticosteroids, eteplirsen, golodirsen, cardiovascular medication, and others. Prednisone and deflazacort are the most common types of corticosteroids prescribed for MD. These drugs work by strengthening the muscles, thus delaying the progression of certain types of MD. Eteplirsen is recommended for children suffering from DMD. Corticosteroids are the most commonly prescribed drugs and are thus expected to dominate the market in the forecast period.
By route of administration: The MD market is divided into oral, parenteral, and others. The oral route is one of the most common types of routes due to patient compliance and ease of administration. Corticosteroids are typically administered orally. Parenteral routes include intravenous, intramuscular, or subcutaneous injections. Out of these, IV injections show the fastest effect as the drug is directly injected into the veins.
By distribution channel: The MD market is categorized as hospital pharmacy, online pharmacy, and retail pharmacy. Hospital pharmacies are anticipated to show a higher market share owing to better patient care and access to a range of drugs. Retail pharmacies dispense the medication based on the prescriptions. They may also bloom during the forecast period.
By region: The MD market is segmented into North America, Europe, Asia-Pacific, and LAMEA. The prevalence of MD disease is highest in North America and Europe. Therefore, these segments will dominate the market. Also, several key market players are present in North America, thus fueling the growth of this segment.
Competitive analysis and profiles of the major players in the MD drugs market, such as Bristol Myers Squibb Co., F. Hoffmann La Roche, Fresenius, GlaxoSmithKline, Hikma Pharmaceuticals plc, Mylan Nv, Novartis AG, Pfizer Inc., Sun Pharmaceutical Industries Ltd., and Teva Pharmaceutical Industries Ltd, are provided in this report. Some other key market players include Boehringer Ingelheim and Sanofi.
The muscular dystrophy drugs market is projected to witness strong growth in the upcoming years. This can majorly be attributed to the increase in various experiments and clinical trials conducted by the pharmaceutical industries. These trials focus on the development of potential treatments for MD. One of the most common types of MD, called Duchenne muscular dystrophy (DMD) can be treated with the help of exon-skipping therapy. Exons are part of a DNA that are involved in coding for proteins. This therapy prevents the mutation of the genes, by skipping over the exon responsible for the disease. Since it has shown favorable results, this therapy may also help to drive the market. As there is no permanent cure for MD, available treatments primarily focus on the management of symptoms. The development of more efficacious drugs for the treatment of MD, with fewer side effects, will fuel this market in the upcoming years. There is an unmet need for these drugs, driving the research and development organizations. There has been an increasing prevalence of MD in the last few years. Some factors like increased awareness among the people and better diagnostic methods may also act as a driver for this market.
The MD market may witness some challenges, restricting its growth. Most of the drugs used to treat MD have a high cost. This can act as a barrier for a large group of patients who cannot afford expensive medication. These drugs have limited efficacy that differs from patient to patient. Drugs may be effective in certain types of diseases only. This makes it challenging to find an effective cure for a particular type of MD. Also, there are various regulatory barriers to the development of drugs for rare diseases like this. Since it is a rare disease, a small population is affected by MD. Therefore, there is minimal investment in research and development for this drugs.
The market share for MD drugs is anticipated to witness several new opportunities in the forthcoming years. Novel treatment strategies like the advent of gene therapy techniques and stem cell therapy may provide significant breaks for the key players in the market. Many companies are now attempting to develop innovative strategies and improve the therapies already in use. Because of the rising incidence, the government is interested in funding various research and clinical trials as well as raising public awareness. This has caused an increase in the research and development programs for MD drugs. Owing to these factors, the market is expected to grow during the forecast period.
New product trends to flourish in the market
In August 2020 U.S. FDA approved, Zolgensma, the first gene therapy for the treatment of MD. In recent years, gene therapy has become a promising approach for MD. This treatment makes use of a virus to deliver a copy of a particular gene, namely the SMN1 gene to patients suffering from spinal muscular atrophy. Other than this, various combination therapies are under study for more favorable outcomes. For instance, the combination of exon-skipping therapy with gene therapy is under clinical trials.
Segment overview
By drugs: The MD market is classified as corticosteroids, eteplirsen, golodirsen, cardiovascular medication, and others. Prednisone and deflazacort are the most common types of corticosteroids prescribed for MD. These drugs work by strengthening the muscles, thus delaying the progression of certain types of MD. Eteplirsen is recommended for children suffering from DMD. Corticosteroids are the most commonly prescribed drugs and are thus expected to dominate the market in the forecast period.
By route of administration: The MD market is divided into oral, parenteral, and others. The oral route is one of the most common types of routes due to patient compliance and ease of administration. Corticosteroids are typically administered orally. Parenteral routes include intravenous, intramuscular, or subcutaneous injections. Out of these, IV injections show the fastest effect as the drug is directly injected into the veins.
By distribution channel: The MD market is categorized as hospital pharmacy, online pharmacy, and retail pharmacy. Hospital pharmacies are anticipated to show a higher market share owing to better patient care and access to a range of drugs. Retail pharmacies dispense the medication based on the prescriptions. They may also bloom during the forecast period.
By region: The MD market is segmented into North America, Europe, Asia-Pacific, and LAMEA. The prevalence of MD disease is highest in North America and Europe. Therefore, these segments will dominate the market. Also, several key market players are present in North America, thus fueling the growth of this segment.
Competitive analysis and profiles of the major players in the MD drugs market, such as Bristol Myers Squibb Co., F. Hoffmann La Roche, Fresenius, GlaxoSmithKline, Hikma Pharmaceuticals plc, Mylan Nv, Novartis AG, Pfizer Inc., Sun Pharmaceutical Industries Ltd., and Teva Pharmaceutical Industries Ltd, are provided in this report. Some other key market players include Boehringer Ingelheim and Sanofi.
Muscular Dystrophy Drugs Market Report Highlights
| Aspects | Details |
 By Drugs |
|
 By Route of Administration |
|
 By Distribution Channel |
|
 By Region |
|
 Key Market Players | Hikma Pharmaceuticals plc, Bristol Myers Squibb Co., Mylan Nv, Pfizer Inc., GlaxoSmithKline (GSK), Fresenius, Sun Pharmaceutical Industries Ltd, Teva Pharmaceutical Industries Limited, Novartis AG, F. Hoffmann La Roche |
 | Boehringer Ingelheim, Sanofi |
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