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

Pages: 53 Published: July 29, 2022 Report Code: GDHCOA017

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.

Scope

Overview of NPC, including epidemiology, symptoms, diagnosis, and disease management.

Annualized NPC therapeutics market revenue in 3MM (US, Germany and the UK), annual cost of therapy and treatment usage pattern in 2021 and forecast for 10 years to 2031; forecast covers three time points: base year, 5-year, and 10-year.

Key topics covered include 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. 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.

Key Players

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

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