Niemann-Pick Disease Type C Therapeutics Market

Niemann-Pick Disease Type C Therapeutics Market:Global Industry Analysis 2015 - 2019 and Opportunity Assessment 2020 - 2030

Market Overview

Niemann-Pick disease is a type of lysosomal storage diseases (LSDs). It is a rare genetic disease where intercellular transport of lipid is damaged leading to accumulation of cholesterol glycosphingolipids in various neurovisceral tissues.

The disorder is highly inconsistent as time of onset and symptoms can vary from person to person. It can at times even vary among different members of the same family. Symptoms can develop at any time- perinatal, early infantile, late infantile, juvenile, and adult.

COVID-19 has been observed to affect different populations with different severity. The reason for this could be genetic variability. It will be crucial to study it with regard to understand genetic susceptibility of people suffering from various genetic disorders in order to develop niche markets like Niemann-Pick disease type C therapeutics market.   

Niemann-Pick Disease Type C Therapeutics Market: Drivers and Restraints

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.

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 global market for Niemann-Pick disease type C therapeutics.

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. 

Smaller target population results in small markets which can slow the growth of the market of Niemann-Pick disease type C therapeutics. Unawareness and limited financial resources are other limiting factors in development of the Niemann-Pick disease type C therapeutics market.

Niemann-Pick Disease Type C Therapeutics Market: Overview

The Niemann-Pick disease type C therapeutics market is about see steady growth as 1 in 7100-7700 live births is the combined prevalence of LSDs worldwide.

Based on therapy type enzyme replacement therapy is estimated to register a robust growth in Niemann-Pick disease type C therapeutics market because presently it is the most used therapy out of all FDA approved therapies. Based on distribution channel hospital pharmacies are estimated to lead the growth in Niemann-Pick disease type C therapeutics market.

Niemann-Pick Disease Type C Therapeutics Market: Region-wise Outlook

Approximately 25 million people in the USA suffer from 7000 rare diseases. Consequently it can be estimated that North America will continue to dominate Niemann-Pick disease type C therapeutics market.

Europe can be expected to remain the second lucrative region for Niemann-Pick disease type C therapeutics market owing to the prevalence of LSDs in Western and Scandinavian region of the continent.

With increasing prevalence of LSDs in Middle East due to increased degree of consanguinity this region can be potentially emerging market in the Niemann-Pick disease type C therapeutics market.

Niemann-Pick Disease Type C Therapeutics Market: Key Players

The key players operating in the Niemann-Pick disease type C therapeutics market are

  • Sanofi Genzyme
  • Actelion (Johnsons and Johnsons)
  • Aldagen (Cytomedix)
  • Alexion
  • Okklo Life Sciences
  • Cyclo Therapeutics Inc.
  • Orphazyme
  • Vtesse Inc. (Cydan)
  • Mallinckrodt Pharmaceuticals
  • IntraBio and Merck.

The research report presents a comprehensive assessment of the market and contains thoughtful insights facts historical data and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies.

The research report provides analysis and information according to market segments such as geographies application and industry.

The report covers exhaust analysis on: 

  • Market Segments
  • Market Dynamics
  • Market Size
  • Supply & Demand
  • Current Trends/Issues/Challenges
  • Competition & Companies involved
  • Indication
  • Value Chain

Regional analysis includes:

  • North America (U.S. Canada)
  • Latin America (Mexico. Brazil)
  • Europe (Germany U.K France Italy Spain)
  • South Asia (India ASEAN Countries)
  • East Asia (Japan China & South Korea)
  • Oceania (Australia & New Zealand)
  • Middle East and Africa (GCC Countries South Africa Northern Africa)

The report is a compilation of first-hand information qualitative and quantitative assessment by industry analysts inputs from industry experts and industry participants across the value chain. The report provides in-depth analysis of parent market trends macro-economic indicators and governing factors along with market attractiveness as per segments.

The report also maps the qualitative impact of various market factors on market segments and geographies.

Report Highlights:

  • Detailed overview of parent market
  • Changing market dynamics in the industry
  • In-depth market segmentation
  • Historical current and projected market size in terms of volume and value
  • Recent industry trends and developments
  • Competitive landscape
  • Strategies of key players and products offered
  • Potential and niche segments geographical regions exhibiting promising growth
  • A neutral perspective on market performance
  • Must-have information for market players to sustain and enhance their market footprint

Niemann-Pick Disease Type C Therapeutics Market: Segmentation

Niemann-Pick disease type C therapeutics market is segmented based on drug class, therapy type, distribution channel and region.

drug class

  • Lovastatin
  • Cholestyramine
  • Nicotinic Acid
  • β-cyclodextrin-hydroxypropyl (HBP-CD)
  • Miglustat

therapy type

  • Enzyme Replacement Therapy
  • Small Molecule Therapy
  • Substrate Reduction Therapy

distribution channel

  • Hospital Pharmacies
  • Drug Stores
  • Retail Pharmacies
  • Online Pharmacies

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