GLOBAL MORQUIO SYNDROME (MPS-IV) DRUG MARKET SIZE AND SHARE ANALYSIS - GROWTH TRENDS AND FORECASTS (2023 - 2030)

Global morquio syndrome (MPS-IV) drug market size is expected to reach US$ 4.3 Mn by 2031 from US$ 1.9 Mn in 2024, exhibiting a compound annual growth rate (CAGR) of 12.4% during the forecast period.

Morquio syndrome, also known as mucopolysaccharidosis type IV (MPS IV), is a rare inherited disease characterized by impaired development and short stature. It is caused by a deficiency of the enzyme N-acetylgalactosamine-6-sulfatase which is required for breakdown of glycosaminoglycans. Current treatment options for MPS IV are limited to symptom management and supportive care.

Global Morquio Syndrome (MPS-IV) Drug Market- Regional Insights

• North America is expected to be the largest market for morquio syndrome (MPS-IV) drug market during the forecast period, accounting for over 43.7% of the market share in 2024. The North American region dominates the global morquio syndrome (MPS-IV) drug market with the presence of several top pharmaceutical companies and a large healthcare expenditure.
• Asia Pacific market is expected to be the second-largest market for morquio syndrome (MPS-IV) drug market, accounting for over 25.2% of the market share in 2024. The Asia Pacific region, especially emerging countries like China and India, is witnessing the fastest growth in the morquio syndrome (MPS-IV) drug market. This is due to increasing research and development (R&D) activities, partnerships and agreements among key market players.
• Europe market is expected to be the fastest-growing market for morquio syndrome (MPS-IV) drug market, with a share of 19% during the forecast period. The growth of the market in Europe is emerging as a hotspot at a rapid pace due to favorable policy support, infrastructure developments, and evolving customer preferences.

Figure 1. Global Morquio Syndrome (MPS-IV) Drug Market Share (%), By Region, 2024

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Analyst View:

Global morquio syndrome (MPS-IV) drug market has significant growth potential over the upcoming years. This rare genetic disorder currently has no cure, but recent advancements in gene therapy and enzyme replacement therapies offer hope of effectively treating the condition. Emerging drugs in the pipeline from major pharmaceutical companies are expected to drive the market upon receiving regulatory approvals. Key market players should focus on collaborating with clinical research centers and patient advocacy groups to spread awareness about the condition and available treatment alternatives. Manufacturers still hold opportunities to target developing nations in Asia and Latin America by launching patient assistance programs and differential pricing strategies. Adoption of advanced gene therapies can help address unmet needs of MPS-IV patients more effectively

Global Morquio Syndrome (MPS-IV) Drug Market - Drivers

• Increasing launch of awareness campaigns: Increasing adoption of inorganic growth strategies such as launch of awareness campaigns is expected to drive the market growth over the forecast period. For instance, in May 2019, Takeda Pharmaceutical Company Limited., a global pharmaceutical company, announced that they had launched #MPSAware campaign to coincide with MPS Awareness Day on May 15th 2019. The campaign aims to raise awareness of this group of seven rare genetic diseases and to recognize the patients, carers, and healthcare professionals living and working with MPS (mucopolysaccharidoses) diseases.
• Improving healthcare infrastructure: Improving healthcare infrastructure across the world is set to be a major driver of growth in the global morquio syndrome (MPS-IV) drug market in the near future. The MPS diseases are a group of rare, genetic, metabolic disorders categorized as lysosomal storage diseases (LSDs). As more countries focus on expanding access to advanced healthcare and building out their medical systems, more people around the world will be able to receive effective treatment and management of morquio syndrome. Better access to diagnosis and long-term care will be important for driving long-term patient adherence and compliance to therapies as well. This helps minimize complications and produce the best health outcomes. As public health systems mature in more locations around the globe, they will strengthen the foundations for sustainable growth of the morquio syndrome market in the near future.

Global Morquio Syndrome (MPS-IV) Drug Market - Opportunities

• Emerging markets in Asia Pacific and Latin America: Emerging markets in Asia Pacific and Latin America offer immense potential for growth in global morquio syndrome (MPS-IV) drug market. Morquio syndrome (MPS-IV) is prevalent in developing nations and according to World Health Organization (WHO), countries like India, Brazil, China, and Mexico account for a major share of the global disease burden. These emerging economies are witnessing rapid economic and infrastructural development which is improving access to healthcare. Simultaneously, growing health awareness and expanded insurance coverage are enabling more patients to seek treatment. Pharmaceutical companies can capitalize on these favorable dynamics. Governments in the region have indicated their commitment in tackling non-communicable diseases have launched public health programs that are focused on early detection and management of conditions like morquio syndrome. For instance, the National Medical Mission under India's Ayushman Bharat initiative aims to set up 1.5 million health and wellness centers by 2023, which will boost screening efforts (NITI Aayog, 2020).
• Rising healthcare expenditure: The significant factor influencing the growth rate of the global morquio syndrome (MPS-IV) drug market is the growing healthcare expenditure, which helps in improving its infrastructure. For instance, in June 2020, according to the International Health Care System of the U.S., , S. government organizations aim to improve the healthcare infrastructure by increasing funding, setting legislation, and national strategies, and cofounding and setting basic requirements and regulations for the Medicaid program. Similarly, in November 2022, the Canadian Institute for Health Information reported that the total healthcare expenditure in Canada was US$ 331 Bn in 2022, or US$ 8,563 per Canadian, while health expenditure represented 12.2% of Canada's gross domestic product (GDP) in 2022, following a high of 13.8% in 2020.

Global Morquio Syndrome (MPS-IV) Drug Market - Trends

• Focus on gene therapy and enzyme replacement therapy: The trend of gene therapy and enzyme replacement therapy is having a significant influence on the global morquio syndrome (MPS-IV) drug market. As these innovative therapies provide hope for more effective treatment options, there is growing interest and investment in the development of gene and enzyme therapies for MPS-IV. The promise of more effective therapies than current treatments is reshaping competitive dynamics in the MPS-IV drug market. Traditional enzyme replacement therapies, while helping manage symptoms, cannot cure MPS-IV or stop its progression. Gene therapies and novel enzyme therapies aim to address the underlying genetic defects and could potentially be curative if clinical trials prove successful.

Global Morquio Syndrome (MPS-IV) Drug Market - Restraint

• Lack of skilled healthcare professionals: Low diagnosis rate, lack of awareness, and exorbitant treatment costs are regarded to be limiting factors for market expansion. The condition can only be treated with a small number of drugs; hence cost of care is anticipated to be significant. Further, it is anticipated that high product costs and inadequate reimbursement rules, particularly in the price-sensitive sector, will impede market expansion.

Counterbalance:  The key market players need to increase the awareness of the treatment and also provide proper reimbursement policies, so that the high treatment costs could be easily undertaken by the patients.

• Limited patient pool: Another challenge restricting the market potential is the small patient population. Morquio syndrome is considered an ultra-rare disease with a very low prevalence globally. Recruiting adequate number of patients for clinical trials is difficult due to the limited availability of subjects. As a result, drug developers have to conduct multi-country trials to attain sufficient statistical power. This increases costs, complexity, and development timelines, thus discouraging investments. Moreover, drug efficacy is difficult to establish for rare diseases with the restricted patient samples. The low commercial viability due to small addressable market also deters large pharmaceutical players. Targeting orphan indications requires long-term commitment and novel clinical trial designs for overcoming these restraints.

Counterbalance: In conclusion, while growing disease awareness and presence of robust pipeline are stimulating opportunities, prohibitive treatment costs and limited patient pool remain major bottlenecks for the MPS-IV drugs market. However, ongoing collaborative research efforts hold promise to develop more affordable and patient-friendly therapeutic molecules in the near future.

Figure 2. Global Morquio Syndrome (MPS-IV) Drug Market Share (%), By Treatment, 2024

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Recent Developments

In March 2021, JCR Pharmaceuticals Co., Ltd., a pharmaceutical company, announced that the Ministry of Health, Labour and Welfare (MHLW) in Japan has approved IZCARGO (pabinafusp alfa 10 mL, intravenous drip infusion) for the treatment of mucopolysaccharidosis type II (MPS II, or Hunter syndrome). IZCARGO is a recombinant iduronate-2-sulfatase enzyme replacement therapy (ERT) that relies on J-Brain Cargo, a proprietary technology developed by JCR, to deliver therapeutics across the blood-brain barrier (BBB).

Top Companies in Global Morquio Syndrome (MPS-IV) Drug Market

• BioMarin Pharmaceutical Inc.
• Takeda Pharmaceutical Company Limited.
• Pfizer Inc.
• Sanofi
• Novo Nordisk A/S
• Concert Pharmaceuticals, Inc.
• Alexion Pharmaceuticals Inc.
• Novartis AG
• Amgen Inc.
• Sangamo Therapeutics
• JCR Pharmaceuticals Co., Ltd.

Definition: Mucopolysaccharidosis type IV (MPS IV) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). As a result, the molecules build up in different parts of the body and cause various health problems. MPS IV is an inherited disorder. This means it is typically passed down through families. If both parents carry a nonworking copy of a gene related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease. This is called an autosomal recessive trait.