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 - Trends, Analysis, and Forecast till 2032

Niemann-Pick Disease Type C Treatment Market accounted for USD 2.2 Billion in 2022 and is estimated to be USD 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:

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
• 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.