[2021] Niemann Pick Disease Type C Market Size Is Anticipated To Reach US$ 378.8 Million at a CAGR of 42.3% By 2027, Finds Coherent Market

SEATTLE, April 14, 2021, (PHARMIWEB) — Global Niemann-Pick disease type C Treatment Market

Overview:

Niemann-Pick disease type C falls under the group of lysosomal storage disorders. The symptoms can begin at different ages. It can be categorised as: juvenile (6 to 15 years), infantile (after birth to less than 6 years), and adults (15 years to greater). In new-borns, NPC leads to various disorders such idiopathic neonatal cholestasis, Gaucher disease, tyrosinemia, alpha-1-antitrypsin deficiency, and various congenital infections. NPC leads to metabolic problems, in juvenile, such as GM2 gangliosidosis, lysosomal storage diseases, organic acidemias, maple syrup urine disease, learning disabilities, and attention deficit hyperactivity disorder (ADHD), and certain mitochondrial disorders.

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Drivers:

Key players in the market are focused on obtaining special status for medicines for the treatment of rare diseases, which, in turn, is expected to drive growth of the global Niemann-Pick infection type C treatment market during the forecast period. For instance, In May 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. The company also received Orphan Drug Designation in both the United States and Europe and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration for Trappsol(R) Cyclo(TM).

Moreover, key players in the market are focused on submitting new drug application to the regulatory authorities to launch drugs in the market, which is also expected to augment the growth of global Niemann-Pick infection type C treatment market. For instance, on November 11, 2020, Orphazyme A/S submitted the marketing authorization application to the European Medicine Agency (EMA) for Arimoclomol, for the treatment of Niemann-Pick disease type C. Moreover, 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.

Covid Impact:

The emergence of COVID-19 (SARS-CoV-2) is expected to hinder growth of the global Niemann-Pick disease type C treatment market over the forecast period. As per the reports of 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.

Moreover, in March 2020, the World Health Organization reported that COVID-19 transmission takes place through respiratory droplets (>5-10 μm) and contact routes. Moreover, according to National Centre for Biotechnology Information, May 02, 2020, the infection and the complication increases if the patient has underlying co-morbidities such as elevated levels of liver enzymes, severe respiratory infections, >5-10 μ enlarged liver and spleen (hepatosplenomegaly) etc., which are also the symptoms for NPC.

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Restraints:

Lack of awareness among people about the symptoms of the disease and lack of screening or diagnostic tests for NPC are expected to hamper growth of the global Niemann-Pick diseases type C treatment market. For instance, according the National Centre for Biotechnology Information (NCBI), in 2019, a lot of patients suffering from NPC are not treated on proper time or the doctors are not aware of the symptoms of the diseases, which is expected to hinder growth of the global Niemann-Pick infection type C treatment market.

Segmentation

By Disease Type

• Niemann-Pick Diseases Type C1
• Niemann-Pick Diseases Type C2

By Age Group

• Infantile
• Juvenile
• Adult

By Region

• North America
• Latin America
• Europe
• Asia Pacific
• Middle East, and Africa

Regional Analysis:

North America and Europe are expected to witness substantial growth in the global Niemann-Pick disease type C treatment market due to increasing research and development activities in the region. For instance, in July 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.

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Competitive Landscape:

Key players active in the global Niemann-Pick diseases type C treatment market are 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.

In September 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.

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