The Acid Sphingomyelinase Deficiency market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Acid Sphingomyelinase Deficiency (ASMD): An Overview
Acid sphingomyelinase deficiency (ASMD) is a rare progressive genetic disorder that results from a deficiency of the enzyme acid sphingomyelinase, which is required to break down (metabolize) a fatty substance (lipid) called sphingomyelin that is inherited in an autosomal recessive pattern.
Acid sphingomyelinase, E.C. 3.1.4.12, (ASM) is a lysosomal phosphodiesterase enzyme that hydrolyzes sphingomyelin, a phospholipid storage substance found in the brain, liver, lungs, spleen, and lymph nodes, to ceramide and phosphorylcholine. It is also known as Niemann– Pick disease type A and B.
Acid Sphingomyelinase Deficiency (ASMD) Market Key Facts
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According to the NORD, the exact incidence and prevalence of the disorder are unknown but have been estimated at 1 in 250,000 individuals in the general population.
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As per the study published by Margaret et al., the incidence of ASMD at approximately 0.5 per 100,000 births; estimates extrapolated from the results of carrier screening suggest that the true incidence may be higher in selected populations.
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According to the study published by Giugliani et al., ASMD has an autosomal recessive pattern of inheritance, and the birth prevalence is estimated at 0.4–0.6 per 100,000.
Acid Sphingomyelinase Deficiency (ASMD) Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Acid Sphingomyelinase Deficiency market size by analyzing the impact of current and emerging pipeline therapies. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Acid Sphingomyelinase Deficiency market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MoA), Route of Administration (RoA), molecule types, competition with other therapies, brand value, and their impact on the market.
Acid Sphingomyelinase Deficiency (ASMD) Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and and views of key opinion leaders.
Acid Sphingomyelinase Deficiency (ASMD) Epidemiology Segmentation-
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Total Prevalent cases of Acid sphingomyelinase deficiency
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Diagnosed cases of Acid sphingomyelinase deficiency
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Diagnosed Cases of Acid sphingomyelinase deficiency by clinical phenotype
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Diagnosed Cases of Acid sphingomyelinase deficiency based on clinical manifestations
Acid Sphingomyelinase Deficiency (ASMD) 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 expected to get launched during the study period. The analysis covers Acid Sphingomyelinase Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug.
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 analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Acid Sphingomyelinase Deficiency (ASMD) Therapeutics Analysis
On August 31, 2022, the US FDA approved French pharma major Sanofi’s Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency (ASMD). With the approval from FDA, Xenpozyme became the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD. The approval was based on positive data from the ASCEND and ASCEND-Peds clinical trials.
Olipudase Alpha is a recombinant human acid sphingomyelinase-based ERT that targets the non-neurological manifestations of acid sphingomyelinase deficiency (ASMD), also known as Niemann–Pick B disease.
Since Sanofi is the only company actively working in the ASMD therapeutics market, it presents an opportunity for other pharma companies to capture the highly available untapped market.
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Acid Sphingomyelinase Deficiency Competitive Intelligence Analysis
4. Acid Sphingomyelinase Deficiency Market Overview at a Glance
5. Acid Sphingomyelinase Deficiency Disease Background and Overview
6. Acid Sphingomyelinase Deficiency Patient Journey
7. Acid Sphingomyelinase Deficiency Epidemiology and Patient Population (In US, EU5, and Japan)
8. Acid Sphingomyelinase Deficiency Treatment Algorithm, Current Treatment, and Medical Practices
9. Acid Sphingomyelinase Deficiency Unmet Needs
10. Key Endpoints of Acid Sphingomyelinase Deficiency Treatment
11. Acid Sphingomyelinase Deficiency Marketed Products
12. Acid Sphingomyelinase Deficiency Emerging Drugs and Latest Therapeutic Advances
13. Acid Sphingomyelinase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Acid Sphingomyelinase Deficiency Market Outlook (In US, EU5, and Japan)
16. Acid Sphingomyelinase Deficiency Access and Reimbursement Overview
17. KOL Views on the Acid Sphingomyelinase Deficiency Market
18. Acid Sphingomyelinase Deficiency Market Drivers
19. Acid Sphingomyelinase Deficiency Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading 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.
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