Niemann-Pick Disease Type C Treatment Market, By Disease Type (Niemann-Pick Diseases Type C1 and Niemann-Pick Diseases Type C2), By Age Group (Infantile, Juvenile, and Adult), and By Region (North America, Europe, Asia Pacific, Latin America, and Middle East & Africa) - Trends, Analysis, and Forecast till 2032

Niemann-Pick Disease Type C Treatment Market accounted for US$ 2.2 billion in 2022 and is estimated to be US$ 4.0 billion by 2032 to grow at CAGR of 6.4%.

Niemann-Pick disease type C (NPC) belongs to a group of lysosomal storage disorders. The onset of symptoms for NPC may begin at different ages. It’s been grouped as: infantile (after birth to less than 6 years), juvenile (6 to 15 years), and adults (15 years to greater). In infants, NPC results in disorders like alpha-1-antitrypsin deficiency, tyrosinemia, Gaucher disease, idiopathic neonatal cholestasis, and various congenital infections. In juvenile, NPC results in metabolic disorders like lysosomal storage diseases, GM2 gangliosidosis, organic acidemias, learning disabilities, and a spotlight deficit hyperactivity disorder (ADHD), syrup urine disease, dopa-responsive dystonia, and certain mitochondrial disorders. In adults, NPC results in Alzheimer disease, dementia, and various psychiatric disorders.

Impact of Covid-19 pandemic on market

COVID-19 pandemic is expected to hamper growth of the Niemann-Pick disease type C treatment market during the forecast period. According to the Indian Journal of Medical Sciences, June 6, 2020, the COVID-19 pandemic has affected the conduction of clinical trials due to unavailability of trial site staff, restrictions for travelling, investigational product availability, and others. These challenges are hindrances for developing new treatment for NPC. This report will quantify the impact of a pandemic on the niemann-pick disease type C treatment market.

Advancements in technology

Novel technologies like next-generation sequencing techniques, facial recognition software to identify rare genetic diseases will spur the market growth in long run which consequently would drive the market for Niemann-Pick disease type C therapeutics. Moreover, regulatory regimes for orphan drugs are often encouraging making investment in R&D for developing niche markets like Niemann-Pick disease type C therapeutics profitable for the firms. Consequently companies can reap benefits like application fee waivers and market protection for longer periods thereby driving the market for Niemann-Pick disease type C therapeutics.

Increasing collaboration between various institutes

Increasing collaboration between various institutes for developing treatment can also boost the Niemann-Pick disease type C therapeutics market. Since onset is in early years diagnosis to exactly determine the disorder becomes challenging. Consequently treatment becomes difficult. Late detection is a major barrier in the Niemann-Pick disease type C therapeutics market. Long gestation periods of clinical trial hamper the growth of markets like Niemann-Pick disease type C therapeutics.

The niemann-pick disease type C treatment market is segmented based on disease type, age group, and region.

On the basis of disease type, the niemann-pick disease type C treatment market is segmented into niemann-pick diseases type C1 and niemann-pick diseases type C2. Based on age group, the market is segmented into infantile, juvenile, and adult.

Regional Insights:

On region the niemann-pick disease type C treatment market is segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America is expected to hold dominant position in the Niemann-Pick disease type C treatment market, owing to the increasing research and development activities for NPC in the region. For instance, on July 17, 2020, Cyclo Therapeutics, Inc. initiated phase III clinical trials for testing the safety and efficacy of the cyclodextrin drugs, Trappsol Cyclo, in NPC patients, after receiving positive results in the phase II clinical trial. Moreover, Europe is also an emerging Niemann-Pick disease type C treatment market, owing to the increasing research and development activities for NPC in this region. For instance, on September 30, 2020, IntraBio Inc. announced positive results for the Phase II clinical trial, which is carried out for testing the safety and efficacy of drug candidate, IB1001, for the treatment of NPC. The clinical trial showed decrease in the severity of ataxia, a symptom of NPC and the trial also passed the endpoint for Clinical Impression of Change in Severity (CI-CS).

Report Scope:

Attribute	Details
Base year for estimation	2021
Forecast period	2022 – 2032
Market representation	Revenue in USD Million & CAGR from 2022 – 2034
Market Segmentation	By Disease Type: Niemann-Pick Diseases Type C1 and Niemann-Pick Diseases Type C2 By Age Group: Infantile, Juvenile, and Adult
Delivery Mode Regional scope	North America - U.S., Canada Europe - UK, Germany, Spain, France, Italy, Russia, Rest of Europe Asia Pacific - Japan, India, China, South Korea, Australia, Rest of Asia-Pacific Latin America - Brazil, Mexico, Argentina, Rest of Latin America Middle East & Africa - South Africa, Saudi Arabia, UAE, Rest of Middle East & Africa
sReport coverage	Revenue forecast, company share, competitive landscape, growth factors, and trends

Segments Covered in the Report:

This report forecasts revenue growth at , regional, and country levels and provides an analysis of the latest industry trends and opportunities in each of the sub-segments from 2022 to 2034. For the purpose of this study, has segmented the niemann-pick disease type C treatment market report based on disease type, age group, and region.

Niemann-Pick Disease Type C Treatment Market, By Disease Type:

• Niemann-Pick Diseases Type C1
• Niemann-Pick Diseases Type C2

Niemann-Pick Disease Type C Treatment Market, By Age Group:

• Infantile
• Juvenile
• Adult

Niemann-Pick Disease Type C Treatment Market, By Region:

• North America
  • Middle East & Africa
    • • GCC
      • Israel
      • South Africa
      • Rest of Middle East & Africa
  • Latin America
    • • Brazil
      • Mexico
      • Rest of Latin America
  • Asia Pacific
    • • China
      • India
      • Japan
      • South Korea
      • Rest of Asia Pacific
  • Europe
    • • Germany
      • UK
      • France
      • Russia
      • Italy
      • Rest of Europe
  • U.S.
  • Canada

The key players operating in the niemann-pick disease type C treatment market includes Orphazyme ApS, CTD Holdings Inc, Neurotrope Inc, Okklo Life Sciences Bv, Intrabio Ltd, Azafaros, Centogene AG Rostock, Actelion Pharmaceuticals Ltd, Mallinckrodt Pharmaceuticals, Merck & Co., Inc., Alexion Pharmaceuticals, Sanofi Genzyme, and Aldagen Inc. The market is especially characterized by collaborations and partnerships between the businesses and acquisitions of smaller players as a part of strategic expansion as well as launching their new product specimen. For instance, In May 18, 2020, CTD Holdings Inc announced that it received fast track designation from the U.S Food and Drug Administration (FDA), for faster testing of the safety and efficacy of Trappsol(R) Cyclo(TM), for the treatment of NPC. Moreover, the company also received Orphan Drug Designation in both the U.S and Europe and Rare Pediatric Disease Designation from the U.S. FDA for Trappsol(R) Cyclo(TM).

• Orphazyme ApS
  • Company Overview
  • Product Portfolio
  • Key Highlights
  • Financial Performance
  • Business Strategies

• CTD Holdings Inc
• Neurotrope Inc
• Okklo Life Sciences Bv
• Intrabio Ltd
• Azafaros
• Centogene AG Rostock
• Actelion Pharmaceuticals Ltd
• Mallinckrodt Pharmaceuticals
• Merck & Co., Inc.
• Alexion Pharmaceuticals
• Sanofi Genzyme
• Aldagen Inc.

1. Research Objective and Assumption
   • Preface
   • Research Objectives
   • Study Scope
   • Years Considered for the study
   • Assumptions
   • Abbreviations
2. Research Methodology
   • Research data
   • Primary Data
     • Primary Interviews
     • Primary Breakdown
     • Key data from Primary Sources
     • Key Thickness Insights
   • Secondary Data
     • Major Secondary Sources
     • Secondary Sources
   • Market Estimation
   • Top-Down Approach
     • Approach for estimating Market Share by Top-Down Analysis (Supply Side)
   • Bottom-Up Approach
     • Approach for estimating market share by Bottom-up Analysis (Demand Side)
   • Market Breakdown and Data Triangulation
   • Research Assumptions
3. Market Preview
   • Executive Summary
   • Key Findings—Global Outlook for Niemann-Pick Disease Type C Treatment Strategies
     • Key Questions this Study will Answer
     • Market Snippet, By Disease Type
     • Market Snippet, By Age Group
     • Market Snippet, By Region
   • Opportunity Map Analysis
   • Executive Summary—3 Big Predictions
4. Market Dynamics, Regulations, and Trends Analysis
   • Market Dynamics
     • Drivers
     • Restraints
     • Market Opportunities
     • Market Trends
   • DR Impact Analysis
   • PEST Analysis
   • Porter’s Five Forces Analysis
   • Opportunity Orbit
   • Market Investment Feasibility Index
   • Macroeconomic Factor Analysis
5. Market Segmentation, By Disease Type, 2020 – 2029, (US$ Mn)
   • Overview
     • Market Value and Forecast (US$ Mn), and Share Analysis (%), 2020 – 2029
     • Y-o-Y Growth Analysis (%), 2020 – 2029
     • Segment Trends
   • Niemann-Pick Diseases Type C1
     • Overview
     • Market Size and Forecast (US$ Mn), and Y-o-Y Growth (%), 2020 – 2029
   • Niemann-Pick Diseases Type C2
     • Overview
     • Market Size and Forecast (US$ Mn), and Y-o-Y Growth (%), 2020 – 2029
6. Market Segmentation, By Age Group, 2020 – 2029, (US$ Mn)
   • Overview
     • Market Value and Forecast (US$ Mn), and Share Analysis (%), 2020 – 2029
     • Y-o-Y Growth Analysis (%), 2020 – 2029
     • Segment Trends
   • Infantile
     • Overview
     • Market Size and Forecast (US$ Mn), and Y-o-Y Growth (%), 2020 – 2029
   • Juvenile
     • Overview
     • Market Size and Forecast (US$ Mn), and Y-o-Y Growth (%), 2020 – 2029
   • Adult
     • Overview
     • Market Size and Forecast (US$ Mn), and Y-o-Y Growth (%), 2020 – 2029
7. Global Market, By Region, 2020 – 2029, (US$ Mn)
   • Overview
     • Market Value and Forecast (US$ Mn), and Share Analysis (%), 2020 – 2029
     • Y-o-Y Growth Analysis (%), 2020 – 2029
     • Regional Trends
   • North America
     • Market Size and Forecast (US$ Mn), By Disease Type, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Age Group, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Country, 2020 – 2029
       • U.S.
       • Canada
   • Europe
     • Market Size and Forecast (US$ Mn), By Disease Type, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Age Group, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Country, 2020 – 2029
       • Germany
       • UK
       • France
       • Russia
       • Italy
       • Rest of Europe
   • Asia Pacific
     • Market Size and Forecast (US$ Mn), By Disease Type, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Age Group, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Country, 2020 – 2029
       • China
       • India
       • Japan
       • South Korea
       • Rest of Asia Pacific
   • Latin America
     • Market Size and Forecast (US$ Mn), By Disease Type, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Age Group, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Country, 2020 – 2029
       • Brazil
       • Mexico
       • Rest of Latin America
   • Middle East & Africa
     • Market Size and Forecast (US$ Mn), By Disease Type, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Age Group, 2020 – 2029
     • Market Size and Forecast (US$ Mn), By Country, 2020 – 2029
       • GCC
       • Israel
       • South Africa
       • Rest of Middle East
8. Competitive Landscape
   • Heat Map Analysis
   • Market Presence and Specificity Analysis
9. Company Profiles
10. Orphazyme ApS
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
11. CTD Holdings Inc
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
12. Neurotrope Inc
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
13. Okklo Life Sciences Bv
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
14. Intrabio Ltd
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
15. Azafaros
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
16. Centogene AG Rostock
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
17. Actelion Pharmaceuticals Ltd
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
18. Mallinckrodt Pharmaceuticals
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
19. Merck & Co., Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
20. Alexion Pharmaceuticals
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
21. Sanofi Genzyme
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
22. Aldagen Inc.
    • Company Overview
    • Product Portfolio
    • Key Highlights
    • Financial Overview
    • Business Strategies
23. The Last Word
    • Future Impact
    • About Us
    • Contact