Mucopolysaccharidosis Treatment Market to Reach $4.37 Billion by 2026; Designation RGX-111 by FDA to Favor Growth

The global mucopolysaccharidosis treatment market size is predicted to reach USD 4.37 billion by 2026. The growing need for the treatment of mucopolysaccharidosis type II (Hunter syndrome) will spur demand for therapies, which in turn, will boost the mucopolysaccharidosis treatment market growth. Moreover, the requirement for cost-effective novel therapies will impel biopharmaceutical companies, thus leading to the expansion of the market. For instance, Elaprase is the most commonly used therapeutic, an enzyme replacement therapy (ERT). The ERT is an expensive therapy option and patients from the developing countries cannot easily access due to high cost restrictions, mentioned in a report, titled “Mucopolysaccharidosis Treatment Market Size, Share & Industry Analysis, By Treatment (Enzyme Replacement Therapy (ERT), and Others), By Disease Type (Mucopolysaccharidosis Type I, Mucopolysaccharidosis Type II, Mucopolysaccharidosis Type IV A, Mucopolysaccharidosis Type VI, and Others), By Route of Administration (Intravenous, Intracerebroventricular (ICV)), By End User (Hospitals, Specialty Clinics, and Others), and Regional Forecast, 2019-2026” the market size stood at USD 1.98 billion in 2018 and is expected to exhibit a CAGR of 10.4% between 2019 to 2026.

Approval of RGX-111 by FDA to Spur Business Opportunities 

RGX-111 (RegenXBio Inc.), a well-studied gene therapy product for the treatment of Hurler syndrome also known as mucopolysaccharidosis type I, received the approval from the FDA. The gene therapy product candidate will be used for the treatment of patients with Hurler syndrome, which in turn, will boost mucopolysaccharidosis treatment market trends. Furthermore, the increasing prevalence of rare genetic disorders will spur new growth opportunities for the market in the forthcoming years. For instance, a genetic disorder is characterized by progressive mental drop, physical functionality loss, impaired language development, corneal and retinal damage, carpal tunnel syndrome and restricted joint movement. In addition, the launch of novel gene therapies by major biopharmaceutical companies will augment healthy growth of the market. For instance, in February 2020 Lysogene, a leading biopharmaceutical company, received the FDA approval for the pipeline candidate of LYS-SAF302 gene therapy in MPS IIIA, also known as the Sanfilippo syndrome.

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High Cost Therapeutics to Hinder Market Expansion

The high cost associated with enzyme replacement therapies (ERT), along with the treatment for MPS is one of the major factors impeding the mucopolysaccharidosis treatment market growth during the forecast period. Moreover, poor diagnosis rates in the developing countries will restrict the growth of the market. The lack of awareness and limited options for the payment of these therapies will also act as dampening factor for the growth of the market. In addition, delays associated with the diagnosis of mucopolysaccharidosis will further hamper the growth of the market. According to Orphanet Journal of Rare Diseases, the study revealed a hold up to decrease the diagnostic delay in the two ultra-orphan diseases of mucopolysaccharidosis types I and mucopolysaccharidosis type III in developing countries.

Focus towards Product Enhancement by Key Player to Propel Business

The mucopolysaccharidosis (MPS) treatment market is led by Shire (now owned by Takeda Pharmaceutical Company Limited). Shire’s main product Elaprase has helped the company to remain in the forefront. For instance, the drug efficiency in terms of treatment outcomes and also its indispensability in treatment of MPS II will spur demand for therapies. Moreover, key players are also focused on enhancing their product portfolio. For instance, BioMarin has the key product offerings of ALDURAZYME, VIMIZIM, and NAGLAZYME in its product portfolio, covering three types of mucopolysaccharidosis.

The Report Lists the Main Companies in the Mucopolysaccharidosis Treatment Market

• Shire (Takeda Pharmaceutical Company Limited)


• Denali Therapeutics


• ArmaGen


• REGENXBIO Inc.


• Sangamo Therapeutics


• BioMarin


• Lysogene

Further Report Findings

• Regional Analysis: The market size in North America stood at USD 1,019.4 million in 2018 and is predicted to witness high growing during the forecast period owing to the adequate reimbursement policies along with presence of major biopharmaceutical companies.


• Leading Segment: The mucopolysaccharidosis type II (Hunter Syndrome) accounted for a share of 35.4%  and is predicted to remain dominant during the forecast period owing to the key products such as Hunterase and Elaprase.

The Mucopolysaccharidosis Treatment Market is Segmented into: