Mucopolysaccharidosis Treatment Market Trends

Growth Drivers

• Innovative drug delivery technologies: The purpose of these technologies effectively act as a vehicle or carrier for a bound or an entrapped therapeutic agent to reach effectively and precisely the actual site of action, thus driving the mucopolysaccharidosis treatment market globally. According to an article published by NLM in June 2023, approximately 30% of comprehensive medical entities and almost 50% of advanced drug compounds are accessible for product manufacture, which are further hydrophobic in nature. In this case, it is suggested to utilize a liquid-based carrier system to bolster the bioavailability of lower water-soluble medications, which have readily grown in recent times.
• Rise in suitable biomarkers: These tend to effectively measure indicators that are present in the body, which are essential for personalized treatment, risk assessment, prognosis, and disease diagnosis, thereby uplifting the mucopolysaccharidosis treatment market. As per the June 2022 JTO report, a clinical study was conducted on 17,513 patients, wherein 83,064 genomic biomarker tests were recorded. Of the total participants, 28.3% to 68.1% received biomarker testing methods, thereby creating a positive outlook for the overall market for patients across different countries.
• Expansion of newborn screening programs: These particular programs are crucial since they are able to identify newborns with critical and treatable disorders prior to symptom appearance, which bolsters the mucopolysaccharidosis treatment market. Additionally, this allows timely intervention that can overcome or diminish long-lasting health issues, as well as developmental risks, thus providing children with a healthy life. As per the May 2023 NLM article, newborn screening in the U.S. organized state-level guidance from the Recommended Uniform Screening Panel (RUSP), which comprised 26 secondary and 35 core disorders, suitable for detecting numerous diagnoses, thereby uplifting the overall market.

2024 Quantitative Outcome of Cell and Gene Therapy Boosting the Mucopolysaccharidosis Treatment Market

Source: Department of Biotechnology

Large-scale Genome Projects Targeting Rare Diseases and Undiagnosed Diseases, Driving the MPS Treatment Market

Source: NLM, October 2022

Challenges

• Supply chain complexities for biologics: The aspect of manufacturing gene therapies and ERTs is extremely complicated, which relies on personalized facilities and a delicate international supply chain for severe raw materials, which negatively impacts the mucopolysaccharidosis treatment market. Besides, effectively maintaining a cold chain for temperature-specific biologics from the factory to patients is logistically expensive and challenging. In addition, any kind of disruption, starting from geopolitical issues to quality control risks in a single facility, can also put a halt to the international production.
• Demonstrating value across ultra-small populations: A part of deliberately conducting powered and robust clinical trials is ethically and scientifically challenging among small populations, which is causing a hindrance in the mucopolysaccharidosis treatment market globally. On the contrary, HTAs frequently dismiss single-arm comparisons and trials to natural data as insufficient, while manufacturers readily struggle to provide high-quality statistical proof, which forces dependency on endpoints and patient-based outcomes, thus creating a gap in the market’s global development.