Acid Sphingomyelinase Deficiency Market: Epidemiology, Therapies, Companies, DelveInsight | Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals

Acid Sphingomyelinase Deficiency therapies, such as XENPOZYME (olipudase alfa), and others, are expected to boost the Acid Sphingomyelinase Deficiency Market in the upcoming years.

DelveInsight has launched a new report on "Acid Sphingomyelinase Deficiency - Market Insights, Epidemiology, and Market Forecast-2034" that 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, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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Some of the key facts of the Acid Sphingomyelinase Deficiency Market Report:

In 2023, the market for acid sphingomyelinase deficiency (ASMD) was valued at approximately USD 120 million, with the United States and Europe accounting for over 90% of the total market share. ASMD, formerly known as Niemann-Pick disease types A, A/B, and B, is a rare, progressively debilitating genetic condition associated with high rates of illness and death.
The estimated number of individuals affected by ASMD is around 2,000 across the United States, Europe (specifically EU4 and the UK), and Japan. In the U.S. alone, the patient population is believed to be under 200, with children representing about two-thirds of these cases.
On November 13, 2024, ClearPoint Neuro, Inc., a global company focused on enabling therapies, announced that the U.S. FDA had granted marketing authorization for its SmartFlow Neuro Cannula via the De Novo pathway. The device is intended for intraputaminal delivery of PTC Therapeutics' gene therapy, KEBILIDITM (eladocagene exuparvovec-tneq), used in the treatment of aromatic L-amino acid decarboxylase (AADC) deficiency. This represents the first FDA-approved device specifically designed to administer gene therapy directly to targeted areas of the brain.
The ASMD market is expected to grow substantially, driven primarily by the emergence of enzyme replacement therapies. In 2022, following regulatory approvals in Japan and Europe, Sanofi's XENPOZYME became the first FDA-approved enzyme replacement therapy for treating non-central nervous system manifestations of ASMD in both pediatric and adult patients.
By 2023, the U.S. accounted for roughly 60% of XENPOZYME's global revenue, underscoring its significant market penetration and the strong demand for treatment within the country. However, challenges such as limited patient enrollment in clinical trials, high development costs, and other barriers hinder the market's progress.
Furthermore, research and development activity in ASMD remains limited, likely due to the condition's rarity, a lack of up-to-date epidemiological data, and persistent gaps in scientific understanding.
The global incidence is estimated at approximately 0.4 to 0.6 cases per 100,000 live births. However, these numbers are likely underreported, indicating that the true incidence may be higher than currently recognized.
Key Acid Sphingomyelinase Deficiency companies such as Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and others are evaluating new drugs for Acid Sphingomyelinase Deficiency to improve the treatment landscape.
Promising Acid Sphingomyelinase Deficiency therapies include XENPOZYME (olipudase alfa), and others.

Acid Sphingomyelinase Deficiency Overview

Acid sphingomyelinase deficiency (ASMD), previously known as Niemann-Pick disease types A, A/B, and B, is a rare inherited autosomal recessive disorder caused by mutations in the SMPD1 gene. These mutations lead to a deficiency of the enzyme acid sphingomyelinase, resulting in the buildup of sphingomyelin within cells. This accumulation predominantly affects the mononuclear phagocyte system, with the liver, spleen, lungs, nervous system, and skeletal system being the most commonly impacted organs.

Acid Sphingomyelinase Deficiency Market Outlook

The therapeutics market for acid sphingomyelinase deficiency (ASMD) is still in its early development, characterized by substantial unmet medical needs and active research initiatives. Although the disease's rarity and the high expenses tied to drug development present notable hurdles, the emergence of innovative treatments and growing awareness provide encouraging prospects for tackling this challenging condition. Ongoing investment in research and development, coupled with strategic partnerships, will be essential for advancing therapeutic options and enhancing outcomes for patients.

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Acid Sphingomyelinase Deficiency Marketed Drugs
XENPOZYME (olipudase alfa): Sanofi

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]
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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Key benefits of the Acid Sphingomyelinase Deficiency Market Report:
Acid Sphingomyelinase Deficiency market report covers a descriptive overview and comprehensive insight of the Acid Sphingomyelinase Deficiency Epidemiology and Acid Sphingomyelinase Deficiency market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).
The Acid Sphingomyelinase Deficiency market report provides insights into the current and emerging therapies.
The Acid Sphingomyelinase Deficiency market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Acid Sphingomyelinase Deficiency market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Acid Sphingomyelinase Deficiency market.

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Table of Contents

1. Report Introduction
2. Executive Summary
3. SWOT analysis
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. 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. Market Access and Reimbursement of Therapies
15. Market drivers
16. Market barriers
17. Appendix
18. Acid Sphingomyelinase Deficiency Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight

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Related Reports:

Acid Sphingomyelinase Deficiency Pipeline Insights, DelveInsight

"Acid Sphingomyelinase Deficiency Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Acid Sphingomyelinase Deficiency market. A detailed picture of the Acid Sphingomyelinase Deficiency pipeline landscape is provided, which includes the disease overview and Acid Sphingomyelinase Deficiency treatment guidelines.

Contact Info:

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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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