Acid Sphingomyelinase Deficiency Market Set for Strong Growth by 2034, Forecasts DelveInsight

DelveInsight's Acid sphingomyelinase deficiency Market Report provides comprehensive insights into the epidemiology and market dynamics across the 7MM.

LAS VEGAS , NV, UNITED STATES, January 30, 2025 /EINPresswire.com/ -- DelveInsight’s “Acid sphingomyelinase deficiency - Market Insight, Epidemiology, And Market Forecast - 2034″ report offers an in-depth understanding of the Acid sphingomyelinase deficiency, historical and forecasted epidemiology as well as the Acid sphingomyelinase deficiency market trends in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan.

To Know in detail about the Acid sphingomyelinase deficiency market outlook, drug uptake, treatment scenario, and epidemiology trends, Click here: Acid sphingomyelinase deficiency Market Forecast

Some of the key facts of the Acid sphingomyelinase deficiency Market Report:
• The global incidence of acid sphingomyelinase deficiency is estimated to range from 0.4 to 0.6 cases per 100K newborns, though these figures are likely underestimated, suggesting the actual incidence may be higher.
• The estimated prevalence of acid sphingomyelinase deficiency is approximately 2,000 patients across the United States, Europe (EU4 and the UK), and Japan.
• Acid sphingomyelinase deficiency affects both males and females equally, with no significant gender disparity in its prevalence.
• Niemann-Pick disease type A typically presents symptoms as early as 3 months of age, while type B has a variable onset, often in early childhood with signs like hepatosplenomegaly and potential lung involvement.
• In 2023, the acid sphingomyelinase deficiency market size was valued at around USD 120 million.
• The US represented the largest market for acid sphingomyelinase deficiency in 2023, with a market size exceeding USD 55 million.
• In Europe, the market size for acid sphingomyelinase deficiency was estimated to be nearly USD 35 million in 2023.
• On November 13, 2024, ClearPoint Neuro, Inc. announced the U.S. FDA granted marketing authorization for its SmartFlow Neuro Cannula, enabling the intraputaminal administration of PTC Therapeutics' gene therapy, KEBILIDI™ (eladocagene exuparvovec-tneq), marking a major milestone in gene therapy delivery.
• Limited pipeline activity in the acid sphingomyelinase deficiency field is attributed to the disorder's rarity, lack of recent epidemiological data, and significant knowledge gaps.
• Key companies involved in the acid sphingomyelinase deficiency market include Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and others.
• The market for acid sphingomyelinase deficiency is expected to see slow growth due to the rare nature of the disease, but advances in gene therapy and ongoing research may drive future developments.

Acid sphingomyelinase deficiency Overview
Acid sphingomyelinase deficiency, formerly recognized as Niemann-Pick disease types A, A/B, and B, is a rare autosomal recessive disorder caused by mutations in the SMPD1 gene, leading to a deficiency in acid sphingomyelinase. This enzyme deficiency results in the accumulation of sphingomyelin within cells, primarily affecting the mononuclear phagocytic system. The liver, spleen, lungs, nervous system, and skeletal system are the most impacted organs.

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Acid sphingomyelinase deficiency Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Acid sphingomyelinase deficiency Epidemiology Segmentation:
The Acid sphingomyelinase deficiency epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by:
• Total Diagnosed Cases of Acid Sphingomyelinase Deficiency
• Total Treated Cases of Acid Sphingomyelinase Deficiency

Download the report to understand which factors are driving Acid sphingomyelinase deficiency epidemiology trends @ Acid sphingomyelinase deficiency Epidemiology Forecast

Acid sphingomyelinase deficiency Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the acid sphingomyelinase deficiency market or that are expected to be launched during the study period. The analysis covers the market uptake of acid sphingomyelinase deficiency by drugs, patient uptake by therapies, and drug sales.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Acid sphingomyelinase deficiency Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Acid sphingomyelinase deficiency Market Strengths
• The approval of olipudase alfa as the first enzyme replacement therapy (ERT) for Acid Sphingomyelinase Deficiency (ASMD) marks a significant breakthrough, offering a disease-modifying treatment for patients.
• Increasing investment in rare disease research and advancements in gene therapy and substrate reduction therapies are driving innovation in the ASMD treatment landscape.

Acid sphingomyelinase deficiency Market Opportunities
• Improved genetic testing and newborn screening programs are facilitating early and accurate diagnosis, leading to better disease management and increased treatment adoption.
• Stronger patient advocacy groups and awareness campaigns are driving better disease recognition, fostering early intervention, and encouraging pharmaceutical investment in ASMD therapies.

Scope of the Acid Sphingomyelinase Deficiency Market Report
• Study Period: 2020–2034
• Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
• Key Acid sphingomyelinase deficiency Companies: Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and others.
• Acid sphingomyelinase deficiency Therapeutic Assessment: Acid sphingomyelinase deficiency current marketed and Acid sphingomyelinase deficiency emerging therapies
• Acid sphingomyelinase deficiency Market Dynamics: Acid sphingomyelinase deficiency market drivers and Acid sphingomyelinase deficiency market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Acid sphingomyelinase deficiency Unmet Needs, KOL’s views, Analyst’s views, Acid sphingomyelinase deficiency Market Access and Reimbursement

To learn more about Acid sphingomyelinase deficiency companies working in the treatment market, visit @ Acid sphingomyelinase deficiency Clinical Trials and Therapeutic Assessment

Table of Contents
1. Acid sphingomyelinase Deficiency Market Report Introduction
2. Executive Summary for Acid sphingomyelinase deficiency
3. SWOT analysis of Acid sphingomyelinase deficiency
4. Acid sphingomyelinase deficiency Patient Share (%) Overview at a Glance
5. Acid sphingomyelinase Deficiency Market Overview at a Glance
6. Acid sphingomyelinase deficiency Disease Background and Overview
7. Acid sphingomyelinase deficiency Epidemiology and Patient Population
8. Country-Specific Patient Population of Acid sphingomyelinase deficiency
9. Acid sphingomyelinase deficiency Current Treatment and Medical Practices
10. Acid sphingomyelinase deficiency Unmet Needs
11. Acid sphingomyelinase deficiency Emerging Therapies
12. Acid sphingomyelinase Deficiency Market Outlook
13. Country-wise acid sphingomyelinase deficiency Market Analysis (2020–2034)
14. Acid sphingomyelinase deficiency Market Access and Reimbursement of Therapies
15. Acid sphingomyelinase deficiency Market Drivers
16. Acid sphingomyelinase deficiency Market Barriers
17. Acid sphingomyelinase deficiency Appendix
18. Acid sphingomyelinase deficiency Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

About DelveInsight
DelveInsight is a leading Healthcare Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate business growth and overcome challenges with a practical approach.

Jatin Vimal
DelveInsight Business Research LLP
+1 469-945-7679
info@delveinsight.com