DelveInsight’s “Acid sphingomyelinase deficiency Market Insight, Epidemiology, and Market Forecast – 2034” report delivers 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, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

 

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Key Takeaways from the Acid Sphingomyelinase Deficiency Market Report

• In December 2024:- Sanofi- A Prospective Observational Study to Assess the Long-term Safety and Immunogenicity of Olipudase Alfa Therapy During Routine Clinical Care in Pediatric Patients Less Than 2 Years of Age With Acid Sphingomyelinase Deficiency. Ancillary protocol-specified procedures to address the study objectives (eg, assessment of ADA) may be considered outside the standard of care for acid sphingomyelinase deficiency (ASMD), but the study methodology remains non-interventional, as the additional collection of data from participants will not dictate treatment.
• The global incidence is estimated to range from 0.4 to 0.6 cases per 100,000 newborns. These figures are likely underestimated, suggesting that the actual incidence may be higher.
• Acid sphingomyelinase deficiency impacts both males and females equally, showing no significant gender disparity in its prevalence.
• Patients with Niemann-Pick disease type A disease develop symptoms as early as age 3 months. Niemann-Pick disease type B has a variable age of presentation but frequently appears early in childhood, when hepatosplenomegaly is detected and symptoms of lung involvement may occur.
• The leading Acid Sphingomyelinase Deficiency Companies such as Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and others.
• Promising Acid Sphingomyelinase Deficiency Therapies such as Olipudase alfa/GZ402665/ Recombinant human acid sphingomyelinase, and others.

 

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Acid Sphingomyelinase Deficiency Epidemiology Segmentation in the 7MM

• Total Diagnosed Cases of Acid Sphingomyelinase Deficiency
• Total Treated Cases of Acid Sphingomyelinase Deficiency

 

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Acid Sphingomyelinase Deficiency Approved Drugs

XENPOZYME (olipudase alfa): Sanofi

XENPOZYME, a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin. In individuals with acid sphingomyelinase deficiency, the deficiency in the ASM enzyme leads to sphingomyelin accumulation in various tissues. XENPOZYME is not expected to cross the blood-brain barrier or modulate CNS manifestations of acid sphingomyelinase deficiency. XENPOZYME has not been studied in patients with acid sphingomyelinase deficiency type A.

 

Acid Sphingomyelinase Deficiency Drugs Market Insights

Enzyme replacement therapy (ERT) for acid sphingomyelinase deficiency is a pivotal treatment that restores deficient sphingomyelinase enzyme activity, thereby mitigating the accumulation of sphingomyelin and alleviating disease symptoms. Administered via intravenous infusion, ERT has been shown to reduce hepatosplenomegaly, improve hematological parameters, and stabilize neurological functions. While effective, ERT requires ongoing administration and is associated with potential side effects such as infusion reactions and allergic responses. The current market includes FDA-approved therapies like olipudase alfa, and research continues to refine treatment protocols and explore combination strategies to enhance patient outcomes and manage long-term treatment challenges.

 

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Acid Sphingomyelinase Deficiency Market Outlook

The Acid sphingomyelinase deficiency therapeutics market remains in its early stages, with significant unmet needs and ongoing research efforts. The rarity of the disease and the high costs associated with drug development pose challenges, but the development of novel therapies and increasing awareness offer promising opportunities for addressing this complex condition. Continued investment in research and development, along with strategic collaborations, will be key to advancing treatment options and improving patient outcomes.

 

Scope of the Acid Sphingomyelinase Deficiency Market Report

• Study Period: 2020-2034
• Coverage: 7MM
• Acid Sphingomyelinase Deficiency Companies: Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and Many Others.
• Acid Sphingomyelinase Deficiency Therapies- Olipudase alfa/GZ402665/ Recombinant human acid sphingomyelinase, 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

 

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Table of Content

1. Key Insights
2. Report Introduction
3. Executive Summary of Acid Sphingomyelinase Deficiency (ASMD)
4. Key Events
5. ASMD Epidemiology and Market Forecast Methodology
6. ASMD Market Overview at a Glance
7. Disease Background and Overview: ASMD
8. Treatment and Management
9. Epidemiology and Patient Population of ASMD in the 7MM
10. Patient Journey
11. Marketed Therapies
12. ASMD: Seven Major Market Analysis
13. Unmet Needs
14. SWOT Analysis
15. KOL Views
16. Market Access and Reimbursement
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight

 

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