Acid Sphingomyelinase Deficiency (ASMD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Acid Sphingomyelinase Deficiency (ASMD) Market Outlook

Thelansis’s “Acid Sphingomyelinase Deficiency (ASMD) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" 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 Acid Sphingomyelinase Deficiency (ASMD) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Acid Sphingomyelinase Deficiency (ASMD) Overview

Acid sphingomyelinase deficiency (ASMD), also known historically as Niemann-Pick disease types A, A/B, and B, is a lysosomal storage disease stemming from inadequate activity of the enzyme acid sphingomyelinase (ASM). This insufficiency results in the buildup of varying sphingomyelin levels within the body. ASMD is an inherited disorder caused by genetic variations that modify specific bodily processes, leading to the onset of the disease. The gene responsible for ASMD is referred to as SMPD1. This condition manifests with foam cell infiltration across different tissues, lipid storage, and a range of clinical symptoms that might overlap. Among these symptoms, pulmonary insufficiency, hepatosplenomegaly, and neurodegeneration stand out. The clinical presentations of ASMD serve as the primary indicators for differentiating this disease, which presents itself as a multi-organ disorder. While hepatosplenomegaly and lung dysfunction are prevalent features in ASMD, ASMD A also involves the central nervous system. ASMD A progresses rapidly and is frequently fatal within the initial years of life. On the other hand, ASMD B demonstrates a slower progression, featuring milder symptoms and often allowing patients to reach adulthood. The diagnosis of ASMD is established by identifying biallelic pathogenic variants in the SMPD1 gene using molecular genetic testing. Additionally, the assessment of residual acid sphingomyelinase enzyme activity, measuring less than 10% of controls, in peripheral blood lymphocytes or cultured skin fibroblasts confirms the diagnosis. Enzyme Replacement Therapy (ERT) utilizing Olipudase alfa (Xenpozyme®) effectively mitigates sphingomyelin accumulation in organs such as the lung, liver, and spleen, excluding the central nervous system.

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: 2021-2032

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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