Morquio Syndrome Market

• The Morquio Syndrome market size is projected to witness sluggish growth throughout the forecast period (2024–2034) due to misunderstanding of the definitions for prevalence and incidence of the condition, poorly reported diagnostic methods as well as patient characteristics, and absence of any suitable quality assessment tool.
• A uncommon genetic birth condition called Morquio syndrome is thought to affect one in every 200,000 babies. The condition may not be evident at birth, and symptoms typically appear between the ages of one and three. The symptoms of Morquio syndrome worsen as a child gets older because it is a progressive illness.
• The leading Morquio Syndrome Companies such as Sangamo Therapeutics, ArmaGen and GREEN CROSS CORP, Concert Pharmaceuticals Inc., Alexion Pharmaceuticals Inc., BioMarin, Genzyme Corporation, Shire, JCR Pharmaceuticals Co., Ltd., Sanofi, Regenxbio, ArmaGen Inc., Lysogene S.A., and others are debveloping therapies for Morquio Syndrome Treatment.

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DelveInsight’s report titled “Morquio Syndrome Market Insights, Epidemiology, and Market Forecast – 2034” comprehensively analyzes Morquio Syndrome, also known as MPS IV or mucopolysaccharidosis IV. The report includes a detailed examination of the historical and projected epidemiology data, including diagnosed prevalent cases, type-specific diagnosed prevalent cases, as well as treatable cases of Morquio Syndrome. The Morquio Syndrome market report offers an in-depth understanding of the various aspects related to the patient population, including diagnosis, prescription patterns, physician perspectives, market access, treatment, and future market developments for the seven major markets, including the United States, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan from 2020 to 2034.

To gauge the market’s overall potential and identify business opportunities, the report discusses current Morquio Syndrome treatment practices and algorithms and the related unmet medical needs.

Morquio Syndrome Treatment Market

Morquio Syndrome Overview

Mucopolysaccharidosis IV (MPS IV) is a mucopolysaccharide storage disease that exists in two forms (MPS IVA and MPS IVB). These are autosomal recessive genetic conditions that comprise a continuum consisting of a severe form with rapid progression and another slowly progressing form. The severe form becomes apparent between the ages of one and three and typically presents with knock-knees and breastbone prominence. The slowly progressing form, which may not become apparent until adolescence, presents with hip pain and stiffness.

Although they are subtypes of the same disease, it is important to distinguish MPS IVA from MPS IVB for the purposes of effective treatment and disease management. MPS IVA can only be distinguished from MPS IVB by molecular genetic or biochemical testing because of the similarities in the symptoms they present. MPS IV occurs because of a deficiency of the enzyme N-acetyl-galactosamine-6-sulfatase (GALNS) and MPS IVB occurs due to a deficiency of beta-galactosidase. The clinical features of MPS IVB are usually fewer and milder than those associated with MPS IVA.

Morquio Syndrome Diagnosis

MPS IV diagnosis is suggested by the findings of medical history, physical examinations, skeletal X-rays, and urine glycosaminoglycans (GAG) analysis. Excessive amounts of keratan sulfate will usually be present in the urine.

Diagnosis of MPS IVA is confirmed by low GALNS enzyme activity in cultured blood or skin cells and/or molecular genetic testing to identify GALNS gene mutations. MPS IVB diagnosis is confirmed by the finding of a beta-galactosidase deficiency in blood or skin cells and/or molecular genetic testing to identify GLB1 gene mutations.

Morquio Syndrome Treatment Landscape

In 2014, the FDA approved a recombinant human GALNS enzyme replacement therapy (elosulfase alfa, or VIMIZIM) for the treatment of MPS IVA. VIMIZIM is manufactured by BioMarin Pharmaceutical Inc. Other treatment of MPS IV is symptomatic and supportive. Surgery to decompress and fuse the bones of the upper neck to the base of the skull can prevent destabilization of the cervical vertebrae and potential damage to the spinal cord.

Morquio Syndrome Epidemiology

The Morquio Syndrome epidemiology section provides insights into the historical and current Morquio Syndrome patient pool and forecasted trends for seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool, its trends, and assumptions undertaken.

The epidemiology section on the Morquio Syndrome market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining the key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

Key Findings

• As per a study by Leadley et al. (2014), point prevalence of Morquio A was 1 per 926,000 in Australia, 1 per 1,872,000 in Malaysia and 1 per 599,000 in UK and Morquio (unclassified) was 1 per 323, 000 in Denmark. Birth prevalence of Morquio A (using recommended diagnostic methods) ranged from 1 per 71,000 in UAE to 1 per 500,000 in Japan.
• According to Matalon & Matalon, 2003, prevalence estimates for Morquio syndrome range from 3 in 1 million persons in the UK.
• According to Leadley et al. (2014), based on the Morquio Syndrome sub-types, the prevalence of Morquio syndrome in the UK is 0.0167 per 10,000, whereas its point prevalence is estimated to be 1 per 599,000.

Morquio Syndrome Market Outlook

The market outlook for Morquio Syndrome does not really indicate a potential growth or increased attention in the coming years. The market is expected to stay stagnant as the recent approval for Morquio Syndrome or MPS IV is only attributable to a single name with neglible activity around the R&D and clinical hurdles imposed for the Morquio Syndrome. Elosulfase alfa is a synthetic version of the enzyme N-acetylgalactosamine-6-sulfatase. It was approved by the FDA in 2014 for the treatment of Morquio syndrome. Elosulfase alfa was developed by BioMarin Pharmaceutical Inc. and is marketed under the brand VIMIZIM.

According to DelveInsight, the Morquio Syndrome market in the 7MM is expected to triviality during the study period 2020–2034.

Morquio Syndrome Drug Chapters

Marketed Morquio Syndrome Drugs

VIMIZIM (elosulfase alfa): BioMarin Biopharmaceuticals

Vimizim (elosulfase alfa / BMN 110) is an enzyme replacement therapy (ERT) for treating patients with Morquio A syndrome (MPS IVA). The drug was developed by BioMarin Pharmaceutical and is the first ERT approved for the treatment of Morquio A syndrome. BioMarin Pharmaceutical received the US FDA approval for treatment of patients with Morquio A syndrome in February 2014. The drug has also obtained orphan drug designation in the US and European Union (EU). The company has also submitted marketing approval applications for the drug in the EU.

Note: Detailed marketed therapies assessment will be provided in the final report...

Emerging Morquio Syndrome Drugs

The currently available treatments for Morquio Syndrome is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). The Morquio Syndrome market dynamics are some what expected to stay unchanged, primarily due to no healthcare spending worldwide.

Note: Detailed emerging therapies assessment will be provided in the final report...

Morquio Syndrome Market Segmentation

DelveInsight’s ‘Morquio Syndrome Market Insights, Epidemiology, and Market Forecast – 2032’ report provides a detailed outlook of the current and future Morquio Syndrome market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Morquio Syndrome Market Size by Countries

The total Morquio Syndrome market size is analyzed for individual countries (the United States Market, EU4 [Germany, France, Italy, and Spain] and the UK market, and Japan). The United States accounted for a larger portion of the 7MM market for Morquio Syndrome in 2022 due to the increasing cases and the cost of treatments.

Morquio Syndrome Market Size by Therapies

The main features of Mucopolysaccharidosis type IV (MPS IV), also known as Morquio syndrome include skeletal defects and possible cardiopulmonary complications. The cost of diagnosing and treating this condition is high, and treatment is not easily available everywhere. As research and development efforts are not really active, it is doubtful that the market size for Morquio Syndrome therapies will expand further, eventually driven by advancements in assessing the risk factors, treatment options, increased awareness, and growing demand for innovative approaches to address the treatment needs of individuals.

Note: Detailed market segment assessment will be provided in the final report...

Morquio Syndrome Drugs Uptake

This section focuses on the sales uptake of potential Morquio Syndrome drugs that have recently launched or are anticipated to be launched in the Morquio Syndrome market between 2020 and 2034. It estimates the market penetration of Morquio Syndrome drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the drug’s probability of success (PoS) in the Morquio Syndrome market.

The emerging Morquio Syndrome therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the Morquio Syndrome market.

Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report on Morquio Syndrome...

Morquio Syndrome Market Access and Reimbursement

DelveInsight’s ‘Morquio Syndrome Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a descriptive overview of the market access and reimbursement scenario of Morquio Syndrome.

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current Morquio Syndrome market trends and fill gaps in secondary findings, we interview KOLs and SMEs working in the Morquio Syndrome domain. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or Morquio Syndrome market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the Morquio Syndrome unmet needs.

Morquio Syndrome : KOL Insights

• DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as the Department of Pediatrics, Saint Louis University, Department of Pediatrics, Gifu University, and many others.
• “When Morquio A patients are treated by ERT, it is likely that urine KS level is reduced rapidly since the enzyme is delivered to kidney and digests the KS stored in kidney. Meanwhile, it is unlikely that blood KS level is reduced in a short term unless the enzyme is delivered to the cartilage directly and improves bone lesions.”
• “The cost of diagnosing and treating this condition is high, and treatment is not easily available everywhere.”

Note: Detailed assessment of KOL Views will be provided in the full report of Morquio Syndrome...

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the Morquio Syndrome Market using various Competitive Intelligence tools, including SWOT analysis, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Morquio Syndrome Pipeline Development Activities

The report provides insights into Morquio Syndrome Clinical Trials within Phase II and III stages. It also analyzes Morquio Syndrome Companies involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging Morquio Syndrome therapies.

Morquio Syndrome Report Insights

• Morquio Syndrome Patient Population
• Morquio Syndrome Therapeutic Approaches
• Morquio Syndrome Pipeline Analysis
• Morquio Syndrome Market Size and Trends
• Morquio Syndrome Market Opportunities
• Impact of Upcoming Morquio Syndrome Therapies

Morquio Syndrome Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• Morquio Syndrome Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Morquio Syndrome Market
• Morquio Syndrome Drugs Uptake

Morquio Syndrome Report Assessment

• Morquio Syndrome Current Treatment Practices
• Morquio Syndrome Unmet Needs
• Morquio Syndrome Pipeline Product Profiles
• Morquio Syndrome Market Attractiveness
• Morquio Syndrome Market Drivers
• Morquio Syndrome Market Barriers

Key Questions Answered In The Morquio Syndrome Market Report

• What are the key findings of the market across the 7MM, and what country will have the largest Morquio Syndrome market size during the forecast period (2024–2034)?
• At what CAGR is the Morquio Syndrome market, and is epidemiology expected to grow in the 7MM during the forecast period (2024–2034)?
• What are the key drivers of growth in the Morquio Syndrome market (2024–2034)?
• What are the most commonly prescribed medications for Morquio Syndrome (2024–2034)?
• Are regulatory or reimbursement challenges affecting the Morquio Syndrome market (2024–2034)?
• Are there any market barriers or challenges hindering the growth of the Morquio Syndrome market (2024–2034)?
• What strategies are pharmaceutical companies adopting to capture a larger share of the Morquio Syndrome market (2024–2034)?