Opportunities for Regional Players in Morquio Syndrome Treatment Market due to Regulatory Changes

Triveni Triveni
Published on Apr 20, 2023

The Global Morquio Syndrome Treatment Market is expected to witness significant revenue growth during the forecast period. The growth can be attributed to various factors such as an increase in the number of products in different stages of R&D and the focus on treating rare diseases. The prevalence of Morquio syndrome is estimated to range from 1/40,000 to 1/200,000 among newborns, as stated by the National Organization for Rare Disease. The disorder affects approximately one in every 5,000 live births and is caused by lysosomal storage disorders, such as MPS IVA and MPS IVB. Both males and females are equally susceptible to MPS IV, which is expected to increase the market for Morquio syndrome treatment over the forecast period.

Morquio syndrome, also known as mucopolysaccharidosis IV (MPS IV), is a group of inherited lysosomal storage disorders. Lysosomes, which are the main digesting organelles in cells, break down certain carbohydrates and fats through enzymes. People with MPS diseases lack or have dysfunctional lysosomal enzymes, causing an abnormal accumulation of complex carbohydrates, such as mucopolysaccharides or glycosaminoglycans, in the arteries, skeleton, eyes, joints, ears, skin, and/or teeth. This accumulation can also occur in the respiratory system, liver, spleen, central nervous system, blood, and bone marrow, leading to gradual damage to cells, tissues, and numerous organ systems of the body.

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Major companies in Morquio Syndrome Treatment Market:

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

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Driving Factors in Morquio Syndrome Treatment Market:

  1. Growing research and development (R&D) activities: The Morquio Syndrome Treatment Market is driven by the increased R&D activities being conducted by various pharmaceutical companies to develop new and innovative treatments for this rare disease.
  2. Increase in the number of products in preliminary and advanced stages of R&D: The number of products in the preliminary and advanced stages of R&D has been increasing, which is expected to drive market growth.
  3. Focus on treatment of rare diseases: There is a growing focus on the treatment of rare diseases, such as Morquio Syndrome, which is driving the growth of the Morquio Syndrome Treatment Market.
  4. Rising prevalence of Morquio Syndrome: The prevalence of Morquio Syndrome is rising globally, which is a key factor driving the demand for treatment options for this rare disease.
  5. Favorable government initiatives: Governments in various regions are initiating programs and policies to support the development and availability of treatments for rare diseases, which is expected to drive market growth.

Restraints in Morquio Syndrome Treatment Market:

  1. Limited Awareness: Lack of awareness about Morquio syndrome among healthcare professionals and patients is a major restraint in the market. This leads to delayed diagnosis and treatment, which can affect the market growth.
  2. High Cost of Treatment: The high cost of treatment is a major challenge for patients and healthcare systems, as many patients require long-term treatment and the cost of medication is expensive. This can limit the market growth.
  3. Limited Availability of Treatment: Morquio syndrome is a rare disease, and there are limited treatment options available. This limits the market growth, as patients may have to rely on off-label use of medications or unproven treatments.
  4. Stringent Regulatory Framework: The regulatory framework for drug approval is complex and time-consuming. This can delay the introduction of new drugs to the market, limiting the treatment options available for patients.
  5. Side Effects of Treatment: Some of the medications used for the treatment of Morquio syndrome have significant side effects, which can affect patient compliance and limit the market growth.

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Notable Innovations in Morquio Syndrome Treatment Market:

  1. Enzyme replacement therapy (ERT): This treatment involves regular infusions of a synthetic version of the missing enzyme, which helps to break down the accumulated mucopolysaccharides in the body.
  2. Gene therapy: This innovative approach involves delivering a functional copy of the missing gene to cells in the body, which can produce the missing enzyme and help to break down the accumulated mucopolysaccharides.
  3. Stem cell transplantation: This treatment involves transplanting healthy stem cells into the body, which can produce the missing enzyme and help to break down the accumulated mucopolysaccharides.
  4. Small molecule therapies: This innovative approach involves the use of small molecules that can help to correct the underlying genetic defect in Morquio Syndrome.
  5. Combination therapies: This approach involves using a combination of different treatments, such as ERT and gene therapy, to provide a more comprehensive and effective treatment for Morquio Syndrome.

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