Niemann-Pick Disease Type C Market to Proliferate by 2032, Predicts DelveInsight | Key Companies in the Market - Azafaros A.G., Cyclo Therapeutics, Inc., ZevraDenmark, Genzyme, IntraBio Inc, Mandos Health

The expected launch of various emerging therapies shall fuel the growth of the Niemann-Pick disease type C market during the forecast period, i.e., 2023–2032. The Niemann-Pick disease type C market size is expected to increase at a significant CAGR

LAS VEGAS, Sept. 19, 2023 /PRNewswire/ -- DelveInsight's Niemann-Pick Disease Type C Market Insights report includes a comprehensive understanding of current treatment practices, Niemann-Pick disease type C emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU-4 (Italy, Spain, France, and Germany), the United Kingdom, and Japan].

Key Takeaways from the Niemann-Pick Disease Type C Market Report

• As per DelveInsight analysis, the Niemann-Pick disease type C market is expected to grow positively at a significant CAGR during the study period (2019–2032).
• Type C1 is by far the more common type, accounting for 95% of cases. As per the National Niemann-Pick Disease Foundation (2021), the epidemiology data on NPC are scarce, although prevalence has been estimated to be approximately 1 in 150,000 live births in Western Europe based on the cases identified over a 15-year period in France, Germany, and the UK.
• Leading Niemann-Pick disease type C companies such as Azafaros A.G., Cyclo Therapeutics, Inc., ZevraDenmark, Genzyme, IntraBio Inc, Mandos Health, and others are developing novel Niemann-Pick disease type C drugs that can be available in the Niemann-Pick disease type C market in the coming years.
• Some key therapies for Niemann-Pick disease type C treatment include AZ-3102, Hydroxypropyl-beta-cyclodextrin, Arimoclomol, GZ402665, IB1001, VTS-270, and others.
• In January 2023, Azafaros received IND approval and is currently conducting Phase II trials to evaluate the safety and pharmacokinetic profile in NPC patients.
• In July 2021, Mandos closed the acquisition of VTS-270. The company states that the FDA has not found the drug to be safe and effective for use to treat NPC1 or for any other use.

Discover which therapies are expected to grab the major Niemann-Pick disease type C market share @ Niemann-Pick Disease Type C Market Report

Niemann-Pick Disease Type C Overview

Niemann-Pick disease type C is a rare progressive hereditary condition defined by the body's inability to transport cholesterol and other fatty substances into cells. This incapacity results in an inappropriate buildup of these chemicals in numerous body tissues including brain tissue. Niemann-Pick disease type C varies according to the age of onset, and symptoms can change from person to person. The visceral, neurological, and mental aspects of this hereditary disease distinguish it. Niemann-Pick disease type C is caused by a mutation in the NPC1 or NPC2 genes and is inherited in an autosomal recessive manner. Niemann-Pick disease type C has a highly variable clinical presentation, with significant heterogeneity in the onset of type C symptoms (enlarged liver, enlarged spleen, or jaundice) and the development of the disease. Niemann-Pick disease type C is diagnosed based on symptoms such as psychiatric disorders, neurologic abnormalities, and so on. This condition is sometimes misinterpreted as another linked neurological disorder.

Niemann-Pick Disease Type C Epidemiology Segmentation

The Niemann-Pick disease type C epidemiology section provides insights into the historical and current Niemann-Pick disease type C patient pool and forecasted trends for individual seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. 

The Niemann-Pick disease type C market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:

• Total Niemann-Pick Disease Type C Prevalent Cases
• Total Niemann-Pick Disease Type C Diagnosed Prevalent Cases 
• Niemann-Pick Disease Type C Gene-specific Diagnosed Prevalent Cases 
• Niemann-Pick Disease Type C Treated Cases

Niemann-Pick Disease Type C Treatment Market 

Niemann-Pick disease type C treatment necessitates the collaborative efforts of a team of professionals. Pediatricians, neurologists, ophthalmologists, pulmonologists, gastroenterologists, and other healthcare providers may be required to arrange a child's therapy systematically and comprehensively. Psychosocial assistance for the entire family is also vital. Affected individuals and their families benefit from genetic counseling. Current Niemann-Pick disease type C treatment focuses on the specific symptoms that each person is experiencing. Niemann-Pick disease type C has no cure, however, patients can benefit from palliative therapies. Occupational therapy can aid in the improvement of posture, speech, and movement. Physical therapy is a crucial element of treatment that can help you keep your mobility for as long as possible.

ZAVESCA/BRAZAVES [miglustat], a substrate reduction medication created by Actelion Pharmaceuticals, is the sole approved medicine in Europe and Japan. The FDA denied its approval for the treatment of NPC due to a lack of data. The US FDA wanted further data, which would take years to obtain and would be nearly difficult to meet the FDA's high standards, owing to the restricted or lack of patient population that could be enrolled in the studies. However, because the medicine is already approved in the United States for the treatment of Gaucher's disease, it is being prescribed to NPC patients as an off-label therapy. Miglustat inhibits the synthesis of glycosphingolipids, one of the chemicals that build in the brains of people with NPC, and hence slows the progression of neurological symptoms. However, this medication is not appropriate for every affected individual.

To know more about Niemann-Pick disease type C treatment, visit @ Niemann-Pick Disease Type C Treatment Drugs 

Key Niemann-Pick Disease Type C Therapies and Companies

• AZ-3102: Azafaros A.G.
• Hydroxypropyl-beta-cyclodextrin: Cyclo Therapeutics, Inc.
• Arimoclomol: ZevraDenmark
• GZ402665: Genzyme
• IB1001: IntraBio Inc
• VTS-270: Mandos Health

Learn more about the FDA-approved drugs for Niemann-Pick disease type C @ Drugs for Niemann-Pick Disease Type C Treatment 

Niemann-Pick Disease Type C Market Dynamics

The Niemann-Pick disease type C market has historically been limited due to the rarity of the disease, several significant dynamics have been shaping its evolution. One of the most notable dynamics is the increasing awareness and diagnosis of NPC. Advances in genetic testing and improved understanding of the disease have led to earlier and more accurate diagnoses. This has driven a growing demand for therapies, as early intervention can significantly improve patient outcomes.

Furthermore, research and development in the field of NPC have seen promising progress. Pharmaceutical companies and research institutions have been actively exploring novel therapeutic approaches, including enzyme replacement therapies, substrate reduction therapies, and gene therapies. This has attracted investment and attention to the Niemann-Pick disease type C market, with a focus on developing effective treatments to address the unmet medical needs of patients.

Additionally, regulatory agencies have recognized the urgency of addressing rare diseases like NPC. Expedited pathways for drug approvals and orphan drug status designations have facilitated the development and commercialization of potential treatments. These regulatory incentives have encouraged pharmaceutical companies to invest in NPC research and development.

While there are positive dynamics in the Niemann-Pick disease type C market, several significant barriers also exist that can hinder progress. The rarity of NPC poses a substantial challenge for pharmaceutical companies and researchers. The small patient population makes it difficult to conduct large-scale clinical trials, which are essential for drug development.

While there have been regulatory incentives to encourage research and development in rare diseases, navigating the regulatory landscape can still be challenging. Companies must meet rigorous safety and efficacy standards, which can be especially demanding for novel therapies in the Niemann-Pick disease type C market. Despite increasing awareness, NPC remains relatively unknown among the general public and even some healthcare professionals. This lack of awareness can lead to delayed diagnoses and hinder early intervention efforts.

Scope of the Niemann-Pick Disease Type C Market Report

• Therapeutic Assessment: Niemann-Pick Disease Type C current marketed and emerging therapies
• Niemann-Pick Disease Type C Market Dynamics: Conjoint Analysis of Emerging Niemann-Pick Disease Type C Drugs
• Competitive Intelligence Analysis: SWOT analysis and Market entry strategies
• Unmet Needs, KOL's views, Analyst's views, Niemann-Pick Disease Type C Market Access and Reimbursement

Discover more about Niemann-Pick disease type C drugs in development @ Niemann-Pick Disease Type C Clinical Trials

Table of Contents

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SOURCE DelveInsight Business Research, LLP