Table of Content - Global Morquio Syndrome (MPS IV) Drug Market

Mucopolysaccharidosis type IV (MPS IV) is a rare disease in which the body is missing or does not have enough of an enzyme needed to break down long chains of sugar molecules. These chains of molecules are called glycosaminoglycans (formerly called mucopolysaccharides). It is caused by a deficiency in the enzymes N-acetylgalactosamine-6-sulfatase (GALNS) or beta-galactosidase which is responsible for breaking down keratan sulfate and chondroitin sulfate. The accumulation of these sugars in cells, tissues, and organs can lead to skeletal dysplasia, short stature, joint abnormalities, and respiratory issues like airway obstruction, heart defects, hearing loss, and corneal opacity. There is no cure for MPS-IV and the treatment options are limited to symptom management. However, enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) have shown some success in management of symptoms and improvement in quality of life for patients. MPS IV is an inherited disorder. This means, it is typically passed down through families. If both parents carry a nonworking copy of a gene that is related to this condition, each of their children has a 25% (1 in 4) chance of developing the disease. This is called an autosomal recessive trait.

Market Dynamics:

Global morquio syndrome (MPS-IV) drug market is primarily driven by the rising prevalence of this rare genetic disorder, growing awareness about available treatment options and strong pipeline drugs with novel mechanisms of action. However, high cost of therapy due to rarity of disease as well as challenges that are associated with production of recombinant enzymes are some of the factors restraining the market growth. Nonetheless, development of improved treatment modalities, increasing research funding for rare diseases and orphan drug designation providing market exclusivity offers lucrative opportunities for the key market players in this market.

Key features of the study:

This report provides in-depth analysis of the global morquio syndrome (MPS-IV) drug market, and provides market size (US$ Mn) and compound annual growth rate (CAGR %) for the forecast period (2024–2031), considering 2023 as the base year
It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
It profiles key players in the global morquio syndrome (MPS-IV) drug market based on the following parameters – company highlights, products portfolio, key highlights, financial performance, and strategies - Key companies covered as a part of this study include BioMarin Pharmaceutical Inc., Takeda Pharmaceutical Company Limited., Pfizer Inc., Sanofi, Novo Nordisk A/S, Concert Pharmaceuticals, Inc., Alexion Pharmaceuticals Inc., Novartis AG, Amgen Inc., Sangamo Therapeutics, and JCR Pharmaceuticals Co., Ltd.
Insights from this report would allow marketers and the management authorities of the companies to make informed decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
The global morquio syndrome (MPS-IV) drug market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
Stakeholders would have ease in decision-making through various strategy matrices used in analyzing the global morquio syndrome (MPS-IV) drug market

Detailed Segmentation:

Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type
- Solid Dosage Form
  - Tablets
  - Capsules
- Liquid Dosage Form
  - Injectable
  - Others
Global Morquio Syndrome (MPS-IV) Drug Market, By Treatment
- Enzyme Replacement Therapy
- Hematopoietic Stem Cell Therapy
- Gene Therapy
- Others
Global Morquio Syndrome (MPS-IV) Drug Market, By Disease Type
- Morquio A
- Morquio B
Global Morquio Syndrome (MPS-IV) Drug Market, By Distribution Channel
- Hospital Pharmacies
- Online Pharmacies
- Retail Pharmacies
Global Morquio Syndrome (MPS-IV) Drug Market, By Region
- North America
- Europe
- Asia Pacific
- Latin America
- Middle East & Africa
Top Companies in the Global Morquio Syndrome (MPS-IV) Drug Market:
- BioMarin Pharmaceutical Inc.
- Takeda Pharmaceutical Company Limited.
- Pfizer Inc.
- Sanofi
- Novo Nordisk A/S
- Concert Pharmaceuticals, Inc.
- Alexion Pharmaceuticals Inc.
- Novartis AG
- Amgen Inc.
- Sangamo Therapeutics
- JCR Pharmaceuticals Co., Ltd.

Table of Contents

Research Objectives and Assumptions
- Research Objectives
- Assumptions
- Abbreviations
Market Purview
- Report Description
  - Market Definition and Scope
- Executive Summary
  - Market Snippet, By Product Type
  - Market Snippet, By Treatment
  - Market Snippet, By Disease Type
  - Market Snippet, By Distribution Channel
  - Market Snippet, By Region
- Coherent Opportunity Map (COM)
Market Dynamics, Regulations, and Trends Analysis
- Market Dynamics
  - Increasing Launch of Awareness Campaigns
  - Lack of skilled healthcare professionals
  - Rising healthcare expenditure
- Key Highlights
- Regulatory Scenario
- Recent Trends
- PEST Analysis
- PORTER’s Analysis
- Product Launches
- Mergers, Acquisitions, and Collaborations
Global Morquio Syndrome (MPS-IV) Drug Market – Impact of Coronavirus (COVID-19) Pandemic
- COVID-19 Epidemiology
- Supply Side and Demand Side Analysis
- Economic Impact
Global Morquio Syndrome (MPS-IV) Drug Market, By Product Type, 2019-2031, (US$ Mn)
- Introduction
  - Market Share Analysis, 2024 and 2031 (%)
  - Y-o-Y Growth Analysis, 2020 – 2031
  - Segment Trends
- Solid Dosage Form
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
    - Tablets
    - Capsules
- Liquid Dosage Form
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
    - Injectable
    - Others
Global Morquio Syndrome (MPS-IV) Drug Market, By Treatment, 2019-2031, (US$ Mn)
- Introduction
  - Market Share Analysis, 2024 and 2031 (%)
  - Y-o-Y Growth Analysis, 2020 – 2031
  - Segment Trends
- Enzyme Replacement Therapy
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Hematopoietic Stem Cell Therapy
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Gene Therapy
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Others
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
Global Morquio Syndrome (MPS-IV) Drug Market, By Disease Type, 2019-2031, (US$ Mn)
- Introduction
  - Market Share Analysis, 2024 and 2031 (%)
  - Y-o-Y Growth Analysis, 2020 – 2031
  - Segment Trends
- Morquio A
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Morquio B
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
Global Morquio Syndrome (MPS-IV) Drug Market, By Distribution Channel, 2019-2031, (US$ Mn)
- Introduction
  - Market Share Analysis, 2024 and 2031 (%)
  - Y-o-Y Growth Analysis, 2020 – 2031
  - Segment Trends
- Hospital Pharmacies
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Online Pharmacies
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
- Retail Pharmacies
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, 2019-2031,(US$ Mn)
Global Morquio Syndrome (MPS-IV) Drug Market, By Region, 2019-2031, (US$ Mn)
- Introduction
  - Market Share Analysis, By Region, 2024 and 2031 (%)
  - Y-o-Y Growth Analysis, For Region, 2020 –2031
  - Country Trends
- North America
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
    - U.S.
    - Canada
- Europe
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
    - Germany
    - U.K.
    - Spain
    - France
    - Italy
    - Russia
    - Rest of Europe
- Asia Pacific
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
    - China
    - India
    - Japan
    - Australia
    - South Korea
    - ASEAN
    - Rest of Asia Pacific
- Latin America
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
    - Brazil
    - Argentina
    - Mexico
    - Rest of Latin America
- Middle East
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country, 2019-2031,(US$ Mn)
    - GCC Countries
    - Israel
    - Rest of Middle East
- Africa
  - Introduction
  - Market Size and Forecast, and Y-o-Y Growth, By Product Type, 2019-2031,(US$
  - Market Size and Forecast, and Y-o-Y Growth, By Treatment, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2019-2031,(US$ Mn)
  - Market Size and Forecast, and Y-o-Y Growth, By Country/Region, 2019-2031,(US$ Mn)
    - North Africa
    - Central Africa
    - South Africa
Competitive Landscape
- Company Profile
  - BioMarin Pharmaceutical Inc.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Takeda Pharmaceutical Company Limited.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Pfizer Inc.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Sanofi
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Novo Nordisk A/S
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Concert Pharmaceuticals, Inc.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Alexion Pharmaceuticals Inc.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Novartis AG
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Amgen Inc.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - Sangamo Therapeutics
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
  - JCR Pharmaceuticals Co., Ltd.
    - Company Highlights
    - Product Portfolio
    - Key Highlights
    - Financial Performance
    - Strategies
- Analyst Views
Section
- References
- Research Methodology
- About us

*Browse 44 market data tables and 35 figures on “Global Morquio Syndrome (MPS-IV) Drug Market” - Global forecast to 2031

View Our Licence Options

Need a Custom Report?

We can customize every report - free of charge - including purchasing stand-alone sections or country-level reports
Customize Now
Want to Buy a Report but have a Limited Budget?

We help clients to procure the report or sections of the report at their budgeted price. Kindly click on the below to avail
Request Discount