Muscular dystrophy refers to a group of inherited genetic disorders that weaken the muscles over time. There are various types of muscular dystrophies based on the specific gene mutations and the muscles affected. The treatment primarily focuses on managing the symptoms and possible complications. However, with advancements in gene and cell therapies, disease-modifying treatments are emerging. Gene therapy aims to deliver a functional copy of the mutated gene to muscle cells to restore normal function.
The global muscular dystrophy treatment market is primarily driven by the increasing research investments in gene and cell therapies. Companies are developing therapies that target the root cause of different types of muscular dystrophy. For instance, Sarepta Therapeutics' Vyondys 53 is the first FDA-approved therapy targeting the underlying cause of Duchenne muscular dystrophy. Other players are evaluating gene therapies across various dystrophinopathies. The success of these therapies is expected to boost the market significantly over the forecast period.
The Global muscular dystrophy treatment market is estimated to be valued at US$ 2.30 Bn in 2024 and is expected to exhibit a CAGR of 11% over the forecast period 2023 to 2030.
Key Takeaways
Key players operating in the muscular dystrophy treatment are F. Hoffmann-La Roche AG, PTC Therapeutics, Sarepta Therapeutics, Pfizer Inc., and Bristol-Myers Squibb Company.
Muscular dystrophy has a rising prevalence globally. According to Parent Project Muscular Dystrophy estimates, over 300,000 people in the United States and many millions worldwide are affected by some form of muscular dystrophy. This growing patient pool is fueling the demand for effective treatment options.
Pharmaceutical companies are pursuing global expansion strategies. In 2021, Sarepta Therapeutics received approval for Vyondys 53 in Brazil and Israel. Bristol-Myers Squibb is conducting late-stage trials of idebenone for Duchenne muscular dystrophy in multiple geographies to secure early commercialization.
Market Drivers
The increasing research investments in gene and cell therapies by biopharmaceutical companies is a major driver for the muscular dystrophy treatment market. Gene therapies offer the potential to directly target the underlying genetic mutation for different types of muscular dystrophy. Many candidates are in late stages of development which are expected to receive regulatory approvals over the forecast period. This will likely transform the treatment landscape.
Current global tensions and geopolitical instability pose challenges for the growth of the muscular dystrophy treatment market. The ongoing conflict between Russia and Ukraine is disrupting supply chains and trade flows. Many pharmaceutical companies have suspended operations or reduced capacities in Russia while international sanctions have made it difficult to do business in the country. This has indirectly affected clinical trials and product availability in Eastern Europe which has a significant patient population for muscular dystrophy. Rising economic uncertainties due to high inflation and the threat of recession in major markets like the US and Europe could shift focus away from non-communicable diseases to more immediate healthcare needs. This could dampen funding for research in rare disease treatments. Pharmaceutical firms will need to carefully assess currency volatility and trade restrictions while planning investments and operations across different geographical regions to mitigate risks. New drug delivery models and collaborations between companies may help sustain supply in impacted areas.
North America currently dominates the muscular dystrophy treatment market in terms of value. The region accounted for over 40% revenue share in 2024 driven by high diagnosis and treatment rates in the US. Factors such as the presence of advanced healthcare infrastructure, favorable reimbursement policies, growing patient affordability and widespread awareness about muscular dystrophy drive higher adoption of approved drugs. The Asia Pacific region is poised to be the fastest growing market during the forecast period. Rapid economic development, growing healthcare expenditure, rising incidence of muscular dystrophy due to genetic factors and increasing collaborations between local drug makers and global biotech companies are boosting the regional market. Emerging nations like India, South Korea and China offer promising opportunities due to their huge population bases and progressive policy changes to accelerate rare disease treatment access.
Geographical regions where the market is concentrated in terms of value include North America and Western Europe due to better healthcare access and infrastructure support for rare disease patients in major countries within these regions. However, Asia Pacific offers the most lucrative growth opportunities and is projected to witness the fastest CAGR during the forecast period from 2023 to 2030. This can be attributed to rising healthcare expenditure levels, improving access to diagnosis and treatments, evolving public policies to streamline orphan drug development and increasing focus of leading pharmaceutical companies on expanding presence in high potential Asian markets.
However, it is also inherited from parents or caused due to a new mutation.
There are various therapies and drugs to control DMD, however, there is still no cure for duchenne muscular dystrophy.Major driving factors in the global duchenne muscular dystrophy market are the introduction of novel drugs and therapies, disease-modifying therapies, strong support from various companies for drug development and support from patient advocacy groups on the regulatory approval process.
The symptoms include muscle weakness, difficulty in walking and breathing and intellectual disability.
Meanwhile, human clinical trials are also underway to find out effective therapy.Various pharmaceutical companies and organizations are increasingly investing in the research and development activities to come up with an effective treatment for duchenne muscular dystrophy.
Moreover, new therapies aim to repair muscle damage caused due to the absence of dystrophin.
Hospitals pharmacies are expected to be the largest distributors in the duchenne muscular dystrophy market.
