Mucopolysaccharidosis Type VII (MPS VII) Treatment Market By Treatment Type (Enzyme Replacement Therapy, Supportive Care, Gene Therapy), By Distribution Channel (Hospital Pharmacies, Specialty Pharmacies, Others), and Geography

Mucopolysaccharidosis Type VII (MPS VII) Treatment Market size was valued at USD 372.1million in 2023 and is expected to grow at 5.3% CAGR from 2024 to 2030. Mucopolysaccharidosis Type VII (MPS VII), also known as Sly syndrome, is an ultra-rare lysosomal storage disorder caused by a deficiency of the enzyme beta-glucuronidase. This deficiency leads to the accumulation of glycosaminoglycans in various tissues, resulting in progressive cellular damage and multi-organ dysfunction. The global mucopolysaccharidosis type VII  treatment market has been experiencing growth due to several key drivers. Increased awareness among healthcare professionals, advancements in diagnostic techniques, and the development of enzyme replacement therapy (ERT) have significantly contributed to market expansion. The approval of Mepsevii (vestronidase alfa-vjbk) by the FDA in November 2017 and by the EMA in August 2018 marked a milestone in mucopolysaccharidosis type VII  treatment, offering the first specific therapy for this condition. However, the extreme rarity of mucopolysaccharidosis type VII, with an estimated incidence of less than 1 in 250,000 births, results in a very limited patient population.

This rarity can discourage investment in research and development. Additionally, the high cost of treatment and challenges in early diagnosis due to the nonspecific nature of initial symptoms pose obstacles to market growth. Despite these challenges, the mucopolysaccharidosis type VII  treatment market presents several opportunities. The potential for developing gene therapies and other advanced treatment modalities offers promising avenues for market expansion. Increasing collaborations between pharmaceutical companies, research institutions, and patient advocacy groups are likely to drive innovation and expand treatment options. Recent trends in the mucopolysaccharidosis type VII  treatment market include a growing focus on personalized medicine and the exploration of combination therapies. Advancements in newborn screening techniques are also improving early diagnosis and treatment initiation for mucopolysaccharidosis type VII  patients. A recent development in the field occurred in October 2023, when Ultragenyx Pharmaceutical Inc. announced positive long-term data from the Phase 3 study of Mepsevii in mucopolysaccharidosis type VII  patients. The study demonstrated sustained improvements in motor function and reduction in urinary glycosaminoglycans over 144 weeks of treatment, further supporting the efficacy of enzyme replacement therapy in mucopolysaccharidosis type VII.

Mucopolysaccharidosis Type VII (MPS VII) Treatment Market Key Developments:

• In November 2020, the European Commission granted orphan drug designation to Avrobio's AVR-RD-05, a lentiviral gene therapy for mucopolysaccharidosis type VII, based on preclinical data showing promise in treating the condition.
• In July 2022, REGENXBIO Inc. announced the clearance of its Investigational New Drug (IND) application by the FDA for RGX-381, a novel gene therapy for the treatment of mucopolysaccharidosis type VII. The company planned to initiate a Phase I/II clinical trial named COMPASS.