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. 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%.

The report "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 the Middle East & Africa) - Trends, Analysis and Forecast till 2034”.

Key Highlights:

• 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).
• In September 2020, Orphazyme A/S announced that the U.S. Food and Drug Administration (U.S. FDA) accepted the New Drug Application for Arimoclomol, with priority review, which was submitted by the company in May 2020.

Analyst View:

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.

Browse 60 market data tables* and 35 figures* through 140 slides and in-depth TOC on “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 the Middle East & Africa) - Trends, Analysis and Forecast till 2034"

Key Market Insights from the report:        

Niemann-pick disease type C treatment market accounted for US$ 33.0 Million in 2020 and is estimated to be US$ 49.2 Million by 2029 and is anticipated to register a CAGR of 4.3%. The market report has been segmented on the basis of disease type, age group, and region.

• Depending upon disease type, the Niemann-Pick Diseases Type C1 segment is projected to grow at highest CAGR over the forecast period.
• By region, 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.

Competitive Landscape:

The prominent player 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 provides detailed information regarding the industrial base, productivity, strengths, manufacturers, and recent trends which will help companies enlarge the businesses and promote financial growth. Furthermore, the report exhibits dynamic factors including segments, sub-segments, regional marketplaces, competition, dominant key players, and market forecasts. In addition, the market includes recent collaborations, mergers, acquisitions, and partnerships along with regulatory frameworks across different regions impacting the market trajectory. Recent technological advances and innovations influencing the market are included in the report.

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