Summary

The 7 major sanfilippo syndrome markets reached a value of US$ 1.7 Billion in 2023. Looking forward, IMARC Group expects the 7MM to reach US$ 2.5 Billion by 2034, exhibiting a growth rate (CAGR) of 3.53% during 2024-2034.

The Sanfilippo syndrome market has been comprehensively analyzed in IMARC’s new report titled “Sanfilippo Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034”. Sanfilippo syndrome, commonly known as mucopolysaccharidosis type III (MPS III), is caused by a single gene abnormality. It is a hereditary metabolic disorder in which the body is unable to effectively break down long chains of sugar molecules known as mucopolysaccharides or glycosaminoglycans (GAGs). As GAGs accumulate, they cause damage to the cells in which they are stored, resulting in the gradual degeneration of the central nervous system. Sanfilippo syndrome is divided into four subcategories based on its genetic cause: IIIA, IIIB, IIIC, and IIID. The symptoms of Sanfilippo syndrome are not noticeable in newborns at birth and begin to emerge as the children grow. Some of the common symptoms include behavioral problems, communication issues such as delayed speech, sleep disorders, developmental delays, intellectual incapacity, seizures, muscle abnormalities, mildly coarse facial characteristics, an enlarged head and tongue, hernia, and some autistic signs. As the child ages, other symptoms, including hearing and vision impairment, arthritis, recurrent respiratory infections, chronic diarrhea, and organ enlargements, such as those of the liver and spleen, may appear. The doctor does a regular physical examination of the patient to diagnose this indication. Aside from this, additional tests are necessary to identify the condition correctly. These generally include a urine test to determine the number of GAGs and specific molecules, genetic testing, which needs a blood or saliva sample, and a blood enzyme test to validate the results. To identify Sanfilippo syndrome, clinicians may also undertake additional newborn screening on infants.

The increasing prevalence of inherited neurodegenerative lysosomal storage disorders is primarily driving the global Sanfilippo syndrome market. In addition to this, the widespread adoption of enzyme replacement therapy that assists in replacing or generating the missing or deficient enzyme in the body due to which Sanfilippo syndrome occurs is also creating a positive outlook for the market. Moreover, several key players are making extensive investments in conducting clinical trials to encourage the development of advanced medications and novel therapeutics that aid in slowing disease progression while trying to prevent complications. This, in turn, is further acting as a significant growth-inducing factor. Apart from this, the increasing number of specialty clinics owing to the escalating need for specialist care of a higher degree for the treatment and management of the disease is also bolstering the market growth. Additionally, the rising consumer awareness towards Sanfilippo syndrome, which has resulted in the formation of several support organizations aiming to raise funds for the treatment, is further augmenting the global market. Besides this, the introduction of favorable guidelines by various regulatory bodies to facilitate faster approvals of pipeline drugs and clinical trials that encourage the entry of new medications is expected to drive the Sanfilippo syndrome market in the coming years.

IMARC Group’s new report provides an exhaustive analysis of the Sanfilippo syndrome market in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report the United States has the largest patient pool for sanfilippo syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario and unmet medical needs, etc. have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the sanfilippo syndrome market in any manner.

Time Period of the Study

Base Year: 2023
Historical Period: 2018-2023
Market Forecast: 2024-2034

Countries Covered

United States
Germany
France
United Kingdom
Italy
Spain
Japan

Analysis Covered Across Each Country

Historical, current, and future epidemiology scenario
Historical, current, and future performance of the sanfilippo syndrome market
Historical, current, and future performance of various therapeutic categories in the market
Sales of various drugs across the sanfilippo syndrome market
Reimbursement scenario in the market
In-market and pipeline drugs
Competitive Landscape:
This report also provides a detailed analysis of the current sanfilippo syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

Late-Stage Pipeline Drugs

Drug Overview
Mechanism of Action
Regulatory Status
Clinical Trial Results
Drug Uptake and Market Performance

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:
Market Insights

How has the sanfilippo syndrome market performed so far and how will it perform in the coming years?
What are the markets shares of various therapeutic segments in 2023 and how are they expected to perform till 2034?
What was the country-wise size of the sanfilippo syndrome market across the seven major markets in 2023 and what will it look like in 2034?
What is the growth rate of the sanfilippo syndrome market across the seven major markets and what will be the expected growth over the next ten years?
What are the key unmet needs in the market?

Epidemiology Insights

What is the size of the sanfilippo syndrome patient pool (2018-2023) across the seven major markets?
What would be the forecasted patient pool (2024-2034) across the seven major markets?
What are the key factors driving the epidemiological trend of sanfilippo syndrome?
What will be the growth rate of patients across the seven major markets?

Sanfilippo Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

What are the current marketed drugs and what are their market performance?
What are the key pipeline drugs and how are they expected to perform in the coming years?
How safe are the current marketed drugs and what are their efficacies?
How safe are the late-stage pipeline drugs and what are their efficacies?
What are the current treatment guidelines for sanfilippo syndrome drugs across the seven major markets?
Who are the key companies in the market and what are their market shares?
What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the sanfilippo syndrome market?

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Table of Contents

1 Preface

2 Scope and Methodology
2.1 Objectives of the Study
2.2 Stakeholders
2.3 Data Sources
2.3.1 Primary Sources
2.3.2 Secondary Sources
2.4 Market Estimation
2.4.1 Bottom-Up Approach
2.4.2 Top-Down Approach
2.5 Forecasting Methodology

3 Executive Summary

4 Sanfilippo Syndrome - Introduction
4.1 Overview
4.2 Epidemiology (2018-2023) and Forecast (2024-2034)
4.3 Market Overview (2018-2023) and Forecast (2024-2034)
4.4 Competitive Intelligence

5 Sanfilippo Syndrome - Disease Overview
5.1 Introduction
5.2 Symptoms and Diagnosis
5.3 Pathophysiology
5.4 Causes and Risk Factors
5.5 Treatment

6 Patient Journey

7 Sanfilippo Syndrome - Epidemiology and Patient Population
7.1 Epidemiology - Key Insights
7.2 Epidemiology Scenario - Top 7 Markets
7.2.1 Epidemiology Scenario (2018-2023)
7.2.2 Epidemiology Forecast (2024-2034)
7.3 Epidemiology Scenario - United States
7.3.1 Epidemiology Scenario (2018-2023)
7.3.2 Epidemiology Forecast (2024-2034)
7.4 Epidemiology Scenario - Germany
7.4.1 Epidemiology Scenario (2018-2023)
7.4.2 Epidemiology Forecast (2024-2034)
7.5 Epidemiology Scenario - France
7.5.1 Epidemiology Scenario (2018-2023)
7.5.2 Epidemiology Forecast (2024-2034)
7.6 Epidemiology Scenario - United Kingdom
7.6.1 Epidemiology Scenario (2018-2023)
7.6.2 Epidemiology Forecast (2024-2034)
7.7 Epidemiology Scenario - Italy
7.7.1 Epidemiology Scenario (2018-2023)
7.7.2 Epidemiology Forecast (2024-2034)
7.8 Epidemiology Scenario - Spain
7.8.1 Epidemiology Scenario (2018-2023)
7.8.2 Epidemiology Forecast (2024-2034)
7.9 Epidemiology Scenario - Japan
7.9.1 Epidemiology Scenario (2018-2023)
7.9.2 Epidemiology Forecast (2024-2034)

8 Sanfilippo Syndrome - Treatment Algorithm, Guidelines, and Medical Practices
8.1 Guidelines, Management and Treatment
8.2 Treatment Algorithm

9 Sanfilippo Syndrome - Unmet Needs

10 Sanfilippo Syndrome - Key Endpoints of Treatment

11 Sanfilippo Syndrome - Marketed Products
11.1 List of Sanfilippo Syndrome Marketed Drugs Across the Top 7 Markets
11.1.1 Drug Name – Company Name
11.1.1.1 Drug Overview
11.1.1.2 Mechanism of Action
11.1.1.3 Regulatory Status
11.1.1.4 Clinical Trial Results
11.1.1.5 Sales Across Major Markets

Kindly note that the complete list of marketed drugs has been provided in the report.

12 Sanfilippo Syndrome - Pipeline Drugs
12.1 List of Sanfilippo Syndrome Pipeline Drugs Across the Top 7 Markets
12.1.1 Trehalose - Seelos Therapeutics
12.1.1.1 Drug Overview
12.1.1.2 Mechanism of Action
12.1.1.3 Clinical Trial Results
12.1.1.4 Safety and Efficacy
12.1.1.5 Regulatory Status
12.1.2 Olenasufligene Relduparvovec - Lysogene
12.1.2.1 Drug Overview
12.1.2.2 Mechanism of Action
12.1.2.3 Clinical Trial Results
12.1.2.4 Safety and Efficacy
12.1.2.5 Regulatory Status
12.1.3 Tralesinidase Alpha - BioMarin Pharmaceutical
12.1.3.1 Drug Overview
12.1.3.2 Mechanism of Action
12.1.3.3 Clinical Trial Results
12.1.3.4 Safety and Efficacy
12.1.3.5 Regulatory Status
12.1.4 DNL126 - Denali
12.1.4.1 Drug Overview
12.1.4.2 Mechanism of Action
12.1.4.3 Clinical Trial Results
12.1.4.4 Safety and Efficacy
12.1.4.5 Regulatory Status
12.1.5 UX 111 - Abeona Therapeutics; Ultragenyx Pharmaceutical
12.1.5.1 Drug Overview
12.1.5.2 Mechanism of Action
12.1.5.3 Clinical Trial Results
12.1.5.4 Safety and Efficacy
12.1.5.5 Regulatory Status

Kindly note that the above only represents a partial list of pipeline drugs and the complete list has been provided in the report.

13. Sanfilippo Syndrome - Attribute Analysis of Key Marketed and Pipeline Drugs

14 Sanfilippo Syndrome - Market Scenario
14.1 Market Scenario - Key Insights
14.2 Market Scenario - Top 7 Markets
14.2.1 Sanfilippo Syndrome - Market Size
14.2.1.1 Market Size (2018-2023)
14.2.1.2 Market Forecast (2024-2034)
14.2.2 Sanfilippo Syndrome - Market Size by Therapies
14.2.2.1 Market Size by Therapies (2018-2023)
14.2.2.2 Market Forecast by Therapies (2024-2034)
14.3 Market Scenario - United States
14.3.1 Sanfilippo Syndrome - Market Size
14.3.1.1 Market Size (2018-2023)
14.3.1.2 Market Forecast (2024-2034)
14.3.2 Sanfilippo Syndrome - Market Size by Therapies
14.3.2.1 Market Size by Therapies (2018-2023)
14.3.2.2 Market Forecast by Therapies (2024-2034)
14.3.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.4 Market Scenario - Germany
14.4.1 Sanfilippo Syndrome - Market Size
14.4.1.1 Market Size (2018-2023)
14.4.1.2 Market Forecast (2024-2034)
14.4.2 Sanfilippo Syndrome - Market Size by Therapies
14.4.2.1 Market Size by Therapies (2018-2023)
14.4.2.2 Market Forecast by Therapies (2024-2034)
14.4.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.5 Market Scenario - France
14.5.1 Sanfilippo Syndrome - Market Size
14.5.1.1 Market Size (2018-2023)
14.5.1.2 Market Forecast (2024-2034)
14.5.2 Sanfilippo Syndrome - Market Size by Therapies
14.5.2.1 Market Size by Therapies (2018-2023)
14.5.2.2 Market Forecast by Therapies (2024-2034)
14.5.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.6 Market Scenario - United Kingdom
14.6.1 Sanfilippo Syndrome - Market Size
14.6.1.1 Market Size (2018-2023)
14.6.1.2 Market Forecast (2024-2034)
14.6.2 Sanfilippo Syndrome - Market Size by Therapies
14.6.2.1 Market Size by Therapies (2018-2023)
14.6.2.2 Market Forecast by Therapies (2024-2034)
14.6.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.7 Market Scenario - Italy
14.7.1 Sanfilippo Syndrome - Market Size
14.7.1.1 Market Size (2018-2023)
14.7.1.2 Market Forecast (2024-2034)
14.7.2 Sanfilippo Syndrome - Market Size by Therapies
14.7.2.1 Market Size by Therapies (2018-2023)
14.7.2.2 Market Forecast by Therapies (2024-2034)
14.7.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.8 Market Scenario - Spain
14.8.1 Sanfilippo Syndrome - Market Size
14.8.1.1 Market Size (2018-2023)
14.8.1.2 Market Forecast (2024-2034)
14.8.2 Sanfilippo Syndrome - Market Size by Therapies
14.8.2.1 Market Size by Therapies (2018-2023)
14.8.2.2 Market Forecast by Therapies (2024-2034)
14.8.3 Sanfilippo Syndrome - Access and Reimbursement Overview
14.9 Market Scenario - Japan
14.9.1 Sanfilippo Syndrome - Market Size
14.9.1.1 Market Size (2018-2023)
14.9.1.2 Market Forecast (2024-2034)
14.9.2 Sanfilippo Syndrome - Market Size by Therapies
14.9.2.1 Market Size by Therapies (2018-2023)
14.9.2.2 Market Forecast by Therapies (2024-2034)
14.9.3 Sanfilippo Syndrome - Access and Reimbursement Overview

15 Sanfilippo Syndrome - Recent Events and Inputs From Key Opinion Leaders

16 Sanfilippo Syndrome Market - SWOT Analysis
16.1 Strengths
16.2 Weaknesses
16.3 Opportunities
16.4 Threats

17 Appendix