Niemann-Pick Type C Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021-2031

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Niemann-Pick Type C Market Overview

The Niemann-Pick Type C (NPC) market size was $128.35 million in 2021 and is forecast to achieve a CAGR of more than 3% during the forecast period. Major symptoms of the disease are neurological and developmental disorders, as well as hepatosplenomegaly, jaundice, cholestasis, bleeding disorders, and eventually dysphagia and even respiratory failure in adults. Early diagnosis remains a critically unmet need, with many patients receiving a diagnosis late after the onset of neurological disease and other musculoskeletal disabilities.

Niemann-Pick Type C Market Size

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NPC Market Drivers

The NPC market is expected to have several novel entrants across the forecast period. This will provide a significant opportunity to address the need for long-term efficacious therapies and will achieve uptake among physicians and contribute to significant market growth. Increased education and awareness among physicians will drive up the rate of diagnosis and contribute to a steady increase in market growth.

Classifications in the NPC Market

NPC is classified based on genetic cause and the signs and symptoms of the condition.

Pediatric-onset NPC

This can range from early infantile onset NPC to the more classic presentation of NPC that can present in middle to late childhood.

Adult-onset NPC

It has a similar neurological presentation as pediatric-onset NPC, although it can include specific manifestations. In older adults, psychiatric symptoms can also appear, and this can be misdiagnosed as dementia or psychiatric illness.

Key Pipeline Products in the NPC Market

NPC has 3 products in the late-stage pipeline at Phase III in the 3MM. There are two novel therapies in Phase I as well that focus on the metabolism and removal of excess cholesterol in the cells.

Key Players in the NPC Market

Some of the key players in the NPC market are IntraBio Ltd., Mandos LLC., and Cyclo Therapeutics Inc.

Market Report Overview

Market size (2021) $128.35 million
CAGR >3%
Forecast period 2022-2031
Classifications Pediatric-Onset NPC and Adult-Onset NPC
Key players IntraBio Ltd., Mandos LLC., and Cyclo Therapeutics Inc

Scope

This report provides:

  • Overview of NPC, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized NPC therapeutics market revenue in 3MM, the annual cost of therapy and treatment usage pattern in 2021 and forecast for 10 years to 2031.
  • Key topics including strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the NPC therapeutics market.
  • Pipeline analysis, comprehensive data assessing emerging trends, and mechanisms of action under development for NPC treatment. The most promising candidates in late-stage development are profiled.
  • Analysis of the current and future market competition in the 3MM NPC therapeutics market. Insightful review of the key industry drivers, restraints, and challenges is also discussed. Each trend is independently researched to provide a qualitative analysis of its implications.

Key Highlights

Report deliverables include a PowerPoint report and Excel-based forecast model

Forecast includes three countries: US, Germany and the UK

Forecasts cover three time points: base year, 5-year, and 10-year

The prevalence of Niemann Pick Type C (NPC) is increasing at a steady rate, in line with population growth in the 3MM at an AGR of 0.29%.

Currently, the treatment regimen for NPC in the 3MM is entirely limited to the use of miglustat (Yargesa, Zavesca or generic).

There are several unmet needs in this space, including therapies with low or minimal toxicity and novel therapies with improved efficacy.

The NPC late-stage R&D pipeline is focused on developing a novel calcium channel regulator and two cyclodextrin therapies.

Steady growth is expected throughout the forecast period as the NPC population steadily increases and novel therapies enter the market.

Reasons to Buy

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the NPC therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the NPC therapeutics market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Johnson & Johnson
Piramal Enterprises
IntraBio Ltd.
Mandos LLC
Cyclo Therapeutics Inc.

Table of Contents

Table of Contents (PowerPoint Deck)

1 Executive Summary

1.1 Executive Summary

2 Disease Overview

2.1 Overview of Niemann-Pick Type C (NPC)

2.2 NPC SWOT Analysis

2.3 Classification of NPC

3 Epidemiology

3.1 Diagnosed Prevalent Cases of Niemann-Pick Type C, Men and Women, 2021–31

3.2 Sources and Methodology

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profiles

5 Unmet Needs and Opportunities

5.1 Unmet Needs in NPC

5.2 Therapies With Low or Minimal Toxicities

5.3 Ease of Administration

5.4 Reduced Cost for NPC Treatments

5.5 Therapies With Improved Efficacy

5.6 Miglustat Approval

6 Pipeline Assessment

6.1 NPC Pipeline Overview

6.2 Leading Pipeline Agents

6.3 Product Profiles: Acetylleucine, Adrabetadex, Trappsol Cyclo

6.4 Pipeline Products – Review Designations

6.5 NPC: Clinical Trials (Phase II/III) Overview

7 R&D Strategies

7.1 Trends in Clinical Trial Design in NPC

7.2 Trends in Deal-Making in NPC

8 Market Outlook

8.1 NPC Market Forecast

8.3 Market Drivers and Barriers

9 Appendix

9.1 Primary Research: KOL Information

9.2 About the Authors

10 Contact Us

Frequently asked questions

Niemann-Pick Type C Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021-2031 standard reports
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