Mucopolysaccharidosis Type IIIA (MPS IIIA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Mucopolysaccharidosis Type IIIA (MPS IIIA) Market Outlook

Thelansis’s “Mucopolysaccharidosis Type IIIA (MPS IIIA) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Sanfilippo Syndrome Type A treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Mucopolysaccharidosis Type IIIA (MPS IIIA) Overview

Mucopolysaccharidosis type III (MPS III), part of the mucopolysaccharidoses group, is a lysosomal storage disease characterized by severe and rapid intellectual deterioration. Initial symptoms typically manifest between the ages of 2 and 6 years, presenting with behavioral disorders such as hyperkinesia and aggressiveness, along with intellectual decline, sleep disturbances, and mild dysmorphism. Neurological involvement becomes more pronounced around age 10, leading to losing motor milestones and communication difficulties. Seizures often occur after age 10; some attenuated forms have been reported. Each MPS III subtype (MPS IIIA, MPS IIIB, MPS IIIC, and MPS IIID) results from deficiencies in one of the four enzymes necessary for heparan sulfate (HS) degradation. Specific enzymes responsible for each subtype include heparan sulfamidase for MPS IIIA, alpha-N-acetylglucosaminidase for MPS IIIB, alpha-glucosaminide N-acetyltransferase for MPS IIIC, and N-acetylglucosamine-6-sulfate sulfatase for MPS IIID. The respective genes for these enzymes have been identified (MPS IIIA on 17q25, MPS IIIB on 17q21, MPS IIIC in the pericentromeric region of chromosome 8, MPS IIID on 12q14), with numerous associated mutations. Allogenic bone marrow grafts are contraindicated, as they do not impede mental deterioration, even in pre-symptomatically engrafted patients. Gene therapy is currently being investigated in animal models for MPS IIIA and MPS IIIB subtypes. The complex neurological degradation and multiple complications necessitate a multidisciplinary approach to provide tailored symptomatic treatment.

• The incidence of MPS in the US has been reported as 0.98 per 100,000 live births.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2023-2033

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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