MUCOPOLYSACCHARIDOSIS (MPS) TREATMENT MARKET ANALYSIS

Market Challenges And Opportunities

Global Mucopolysaccharidosis (MPS) Treatment Market- Driver

Increasing product approvals by regulatory authorities for better treatment outcomes

Increasing product approvals by regulatory authorities for better treatment outcomes is expected to propel the market growth over the forecast period. Key players operating in the market are focusing on obtaining approvals for their advanced therapeutics, and this is expected to drive the market growth over the forecast period. For instance, in June 2021, Biomarine Pharmaceuticals Inc. is a global biotechnology company announced developing and commercializing innovative biopharmaceuticals for rare diseases driven by genetic causes. It had received approval for its Vimizim (elosulfase alfa) from the National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

Adoption of inorganic growth strategies by key players

Adoption of inorganic growth strategies by key players such as collaborations and partnerships is expected to drive growth of the global mucopolysaccharidosis (MPS) treatment market over the forecast period. For instance, in May 2020, Ultragenyx Pharmaceutical Inc., which is focused on developing the first-ever approved treatments for rare and ultrarare diseases and moving the rare disease community forward, and Rentschler Fill Solutions, a contract manufacturing organization (CMO) and aseptic fill and finish services expert company, collaborated for the production of injectable Mepsevii (vestronidase alfa), a recombinant form of the human enzyme beta-glucuronidase, to treat patients with mucopolysaccharidosis (MPS) VII, a rare genetic disorder.

Global Mucopolysaccharidosis (MPS) Treatment Market: Restraint

High cost of therapeutics coupled with poor diagnosis

High costs of therapeutics coupled with poor diagnosis are expected to hinder growth of the global mucopolysaccharidosis (MPS) treatment market during the forecast period. Vimizin (elosulfase-alfa) is a treatment for MPS IV (Morquio Syndrome). It was launched in the market at a cost of US$ 380,000 per year. Patients in emerging economies like India cannot afford this treatment because of the high price.

Global Mucopolysaccharidosis (MPS) Treatment Market- Growth Drivers

• Increasing product approvals by regulatory authorities for better treatment outcomes 
• Adoption of inorganic growth strategies by key player

Global Mucopolysaccharidosis (MPS) Treatment Market: Key Trends

Growing development of enzyme replacement therapy products by companies

The growing development of enzyme replacement therapy products by companies is also likely to increase the availability of ERT therapies in the market, further augmenting Mucopolysaccharidosis (MPS) Treatment Market growth. For  instance, in March 2021, the Ministry of Health, Labour and Welfare (MHLW) approved IZCARGO (pabinafusp alfa 10 mL, intravenous drip infusion) for the treatment of MPS II in Japan. It is a recombinant iduronate-2-sulfatase enzyme replacement therapy (ERT) developed using J-Brain Cargo, a proprietary technology developed by JCR, to deliver therapeutics across the blood-brain barrier (BBB).