Acid Sphingomyelinase Deficiency Market

Therapies for Acid Sphingomyelinase Deficiency, including XENPOZYME (olipudase alfa) and others, are anticipated to drive growth in the Acid Sphingomyelinase Deficiency market in the coming years.

DelveInsight has released a comprehensive report titled “Acid Sphingomyelinase Deficiency – Market Insights, Epidemiology, and Market Forecast-2034,” providing an in-depth analysis of Acid Sphingomyelinase Deficiency, its historical and projected epidemiology, and market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Discover about the Acid Sphingomyelinase Deficiency market report

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

• In 2023, the global market for acid sphingomyelinase deficiency (ASMD) was valued at around USD 120 million, with the United States and Europe contributing over 90% of the total market share. ASMD, previously referred to as Niemann-Pick disease types A, A/B, and B, is a rare, progressively debilitating genetic disorder associated with significant morbidity and mortality.

• The estimated number of ASMD patients is approximately 2,000 across the United States, Europe (EU4 and the UK), and Japan. In the U.S., the patient population is believed to be fewer than 200, with children comprising roughly two-thirds of 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. This device is designed for intraputaminal delivery of PTC Therapeutics’ gene therapy, KEBILIDI™ (eladocagene exuparvovec-tneq), for treating aromatic L-amino acid decarboxylase (AADC) deficiency. It is the first FDA-approved device specifically engineered to administer gene therapy directly to targeted brain regions.

• The ASMD market is projected to expand significantly, primarily driven by the advent 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 managing 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, reflecting strong demand and market penetration. However, factors such as limited patient enrollment in clinical trials, high development costs, and other challenges continue to constrain market growth.

• Research and development in ASMD remain limited, largely due to the disease’s rarity, insufficient epidemiological data, and gaps in scientific knowledge. Globally, the incidence is estimated at approximately 0.4 to 0.6 cases per 100,000 live births, although actual numbers may be higher due to underreporting.

• Key companies developing treatments for ASMD include Sanofi Genzyme, Orphazyme, Takeda Pharmaceutical, Actelion Pharmaceuticals, and others. Promising therapies in the pipeline include XENPOZYME (olipudase alfa) and additional emerging candidates.

Acid Sphingomyelinase Deficiency Overview

Acid sphingomyelinase deficiency (ASMD), formerly referred to as Niemann-Pick disease types A, A/B, and B, is a rare autosomal recessive genetic disorder caused by mutations in the SMPD1 gene. These mutations result in a shortage of the enzyme acid sphingomyelinase, leading to the accumulation of sphingomyelin within cells. This buildup primarily impacts the mononuclear phagocyte system, with the liver, spleen, lungs, nervous system, and skeletal system being the most frequently affected organs.

Acid Sphingomyelinase Deficiency Market Outlook

The acid sphingomyelinase deficiency (ASMD) therapeutics market remains in its nascent stages, marked by significant unmet medical needs and active research efforts. While the rarity of the disorder and the high costs associated with drug development pose considerable challenges, the development of innovative therapies and increasing awareness offer promising opportunities for addressing this complex condition. Continued investment in R&D, along with strategic collaborations, will be critical for expanding treatment options and improving patient outcomes.

Discover how the Acid Sphingomyelinase Deficiency market is rising in the coming years @ https://www.delveinsight.com/sample-request/acid-sphingomyelinase-deficiency-asmd-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

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

To know what’s more in our Acid Sphingomyelinase Deficiency report, visit https://www.delveinsight.com/report-store/acid-sphingomyelinase-deficiency-asmd-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Key benefits of the Acid Sphingomyelinase Deficiency Market Report:

1. 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).

2. The Acid Sphingomyelinase Deficiency market report provides insights into the current and emerging therapies.

3. The Acid Sphingomyelinase Deficiency market report provides a global historical and forecasted market covering drug outreach in 7MM.

4. 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.

Got queries? Click here to know more about the Acid Sphingomyelinase Deficiency market Landscape

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

Click here to read more about Acid Sphingomyelinase Deficiency Market Outlook 2034

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.

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.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Kritika Rehani
Email: Send Email
Phone: +14699457679
Address:304 S. Jones Blvd #2432
City: Las Vegas
State: Nevada
Country: United States
Website: https://www.delveinsight.com/