Key Highlights
- In 2022, the market size of Niemann-Pick Disease Type C (NPC) was the highest in the US among the 7MM, accounting for approximately USD 9 million, expected to increase by 2032.
- There are 787 diagnosed cases of Niemann-Pick Disease Type C (NPC) in the 7MM in 2022, of which 42% of patients are attributed to the US.
The Niemann-Pick Disease Type C market report provides current treatment practices, emerging drugs, market size, market share of the individual therapies, and current and forecasted 7MM from 2019 to 2032. The report also covers current Niemann-Pick Disease Type C (NPC) treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Geography Covered
- The United States
- EU4 (Germany, France, Italy, and Spain) and the United Kingdom
- Japan
Niemann-Pick Disease Type C (NPC) Understanding and Treatment Algorithm
Niemann-Pick Disease Type C (NPC) is a progressive, irreversible, pan-ethnic, and chronically debilitating neurovisceral lysosomal lipid storage disease characterized by visceral, neurological, and psychiatric manifestations. It involves the unique abnormalities of intracellular transport of endocytosed cholesterol with sequestration of unesterified cholesterol in lysosomes and late endosomes. It occurs due to the alterations in the trafficking of endocytosed cholesterol. It ultimately results in the accumulation of cholesterol and glycosphingolipids in various neurovisceral tissues.At the cellular level, Niemann-Pick Type C can be caused by two different genetic mutations. A mutation in the NPC1 gene causes type C1 and type C2 is caused by a mutation in the NPC2 gene. NPC1 is found in the majority of cases as compared to NPC2. Although the exact function of NPC1 and NPC2 is still elusive, these proteins function coordinatedly and are involved in the cellular post-lysosomal/late endosomal transport of cholesterol and other molecules. NPC causes a secondary reduction of ASM activity, leading to all three forms of Niemann-Pick Disease
Niemann-Pick Disease Type C (NPC) Diagnosis
NPC diagnosis includes blood tests, neuroimaging tests, genetic tests, and skin biopsy. A progressive vertical supranuclear gaze palsy, gelastic cataplexy, ataxia, dystonia, and dementia strongly suggest the diagnosis.Further details related to country-based variations are provided in the report…
Niemann-Pick Disease Type C (NPC) Treatment
Although no cure yet exists, the treatment of NPC disease has made significant strides in the past decade, from the earliest treatments with statins to the latest with sugars, such as miglustat and cyclodextrin. The management of NPC is based mainly on supportive care. Treatment focuses on managing symptoms with medication for seizures, sedatives for sleep disturbances, physical therapy to maintain mobility, and speech therapy to preserve communication as long as possible. Chest physiotherapy and antibiotics may help prevent regular lung infections. Individuals with the condition need a gastronomy tube for feeding when they can no longer swallow well enough to avoid choking or malnutrition.Miglustat is the first and only specific drug approved for this disease in Europe in 2009 and Japan in 2012; its objective is to alleviate disease symptoms while attenuating neurodegeneration. In the US, the FDA declined to approve this drug in 2010 and called for more data. However, since the drug is already approved for treating Gaucher's disease in the US, it is being prescribed to NPC patients as an off-label therapy.
The rarity of the disease and lack of expertise translate into misdiagnosis, delayed diagnosis, and barriers to adequate care. Niemann-Pick disease is characterized by a highly heterogeneous presentation, making diagnosis difficult and often delayed.
Niemann-Pick Disease Type C (NPC) Epidemiology
As the market is derived using a patient-based model, the Niemann-Pick Disease Type C (NPC) epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalent cases of Niemann-Pick Disease Type C, Gender-specific Diagnosed Prevalent cases of Niemann-Pick Disease Type C, and Distribution of Diagnosed Prevalent Cases of Niemann-Pick disease Type C by type in the 7MM covering, the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.- There were 787 diagnosed prevalent cases of Niemann-Pick Disease Type C (NPC) estimated to have occurred in the 7MM in 2022, of which 335 of the accounted cases were estimated to be from the US alone, and these cases are anticipated to increase during the forecast period.
- The diagnosed prevalent Niemann-Pick Disease Type C (NPC) cases were divided into Gender-specific cases. The Gender-specific diagnosed prevalent cases of Niemann-Pick Disease Type C (NPC) are categorized into Males and Females, with 354 and 433 cases, respectively, in the 7MM in 2022, which will increase by 2032.
- The distribution of diagnosed prevalent cases of Niemann-Pick disease type C by type was categorized into early-infantile (< 2 years), late-infantile (2 to < 6 years), juvenile (6 to < 15 years), and adolescent/adult-onset (=15 years), with 100, 227, 235, and 226 cases in the 7MM in 2022.
Niemann-Pick Disease Type C (NPC) Drug Chapters
The drug chapter segment of the Niemann-Pick Disease Type C (NPC) report encloses a detailed analysis of Niemann-Pick Disease Type C late-stage, mid-stage, and mid-early stage (Phase III, Phase II/III, Phase II) pipeline drugs. It also helps understand the Niemann-Pick Disease Type C clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.Emerging Drugs
Presently four emerging therapies are focused on the treatment of Niemann Pick Disease Type C - Arimoclomol (Zevra Therapeutics), AZ-3102 (Azafaros A.G.), Trappsol(R) Cyclo or Hydroxypropyl-beta-cyclodextrin (Cyclo Therapeutics, Inc.) and IB1001 (N-acetyl-L-leucine) (IntraBio). These emerging therapies will provide much-needed relief for the patients and can significantly change the market dynamics in the 7MM countries during the forecast period (2023-2032) due to the absence of marketed therapies.Niemann-Pick Disease Type C (NPC) Market Outlook
Until some years ago, Niemann-Pick disease treatment was based on anti-epileptics, anticholinergic, or antidepressants to alleviate symptoms, i.e., tremors, dystonia, or seizures. Niemann-Pick disease has no cure, and potential therapies should be addressed. The main goal is to develop treatments to minimize general symptoms and neurodegeneration.Supportive therapy by multidisciplinary specialists treats different individual manifestations shown by Niemann-Pick type C patients. For instance, if a person is showing neurological concerns like spasticity, cataplexy, movement disorders, sleep disturbance, and seizures, then the treatment options are physical therapy, modafinil, trihexyphenidyl, melatonin, and antiseizure medicines are used respectively. If a person has difficulty with speech, speech therapy is usually recommended. Genetic counseling and general supportive care are also crucial for people suffering from Niemann-Pick Disease Type C.
Unfortunately, no treatment has yet proven able to change the actual course of NPC. Meanwhile, MIGLUSTAT is the first and only specific drug approved for this disease in Europe (2009) and Japan (2012); its objective is alleviating disease symptoms while attenuating neurodegeneration. In the US, the FDA declined to approve this drug in 2010 and called for more data. However, since the drug is already approved for treating Gaucher's disease in the US, it is being prescribed to NPC patients as an off-label therapy.
The total market size of Niemann-Pick Disease Type C (NPC) in the 7MM was approximately USD 34 million in 2022 and is projected to increase during the forecast period (2023-2032)
- The market size of Niemann-Pick Disease Type C (NPC) in the 7MM will increase at a CAGR of about 11.79% due to the advancing treatment landscape.
- Among the EU4 countries, Germany, France, and Italy accounted for the maximum market size of USD 5.8, USD 4.6, and USD 4.2 million in 2022, while Spain occupied the bottom of the ladder in the same year with USD 3.2 million.
- Japan accounted for a market size of USD 2 million in 2022, but these dynamics are expected to change in the forecast period.
Niemann-Pick Disease Type C (NPC) Drug Uptake
This section focuses on the uptake rate of the potential drug expected to be launched in the market during the study period 2019-2032. MIGLUSTAT is the first and only specific drug approved for this disease in Europe (2009) and Japan (2012).Further detailed analysis of emerging therapies drug uptake is in the report…
Niemann-Pick Disease Type C (NPC) Pipeline Development Activities
The report provides insights into therapeutic candidates in Phase III, II, and I. It also analyzes key players involved in developing targeted therapeutics.Pipeline Development Activities
The report covers detailed information on designations, collaborations, mergers and acquisitions, licensing, and patent details for Niemann-Pick Disease Type C (NPC) emerging therapies.KOL Views
To keep up with the current market trends, we take KOLs and SMEs opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on Niemann-Pick Disease Type C evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake along with challenges related to accessibility, include Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, US, Professor of Pediatrics, Professor of Medicine, Rutgers New Jersey Medical School, US, and Department of Pediatrics, Jichi Medical University, Tochigi, Japan.The analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers such as the Willink Biochemical Genetics Unit, Royal Manchester Children's Hospital, UK, Neurology Service, Sant Joan de Déu Hospital, Spain, Boston Children's Hospital, US, etc., were contacted. Their opinion helps understand and validate current and emerging therapies and treatment patterns or Niemann-Pick Disease Type C market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Qualitative Analysis
The publisher performed Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.Market Access and Reimbursement
The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.Scope of the Report
- The report covers a segment of key events, an executive summary, and a descriptive overview of Niemann-Pick Disease Type C, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
- Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines has been provided.
- Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
- A detailed review of the Niemann-Pick Disease Type C market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM Niemann-Pick Disease Type C market.
Niemann-Pick Disease Type C (NPC) Report Insights
- Patient Population
- Therapeutic Approaches
- Pipeline Analysis
- Niemann-Pick Disease Type C Market Size and Trends
- Existing and future Market Opportunity
Niemann-Pick Disease Type C (NPC) Report Key Strengths
- Ten Years Forecast
- The 7MM Coverage
- Niemann-Pick Disease Type C Epidemiology Segmentation
- Key Cross Competition
- Drug Uptake and Key Market Forecast Assumptions
Niemann-Pick Disease Type C (NPC) Report Assessment
- Current Treatment Practices
- Unmet Needs
- Pipeline Product Profiles
- Market Attractiveness
- Qualitative Analysis (SWOT Analysis)
Key Questions Answered
Market Insights
- What was the Niemann-Pick Disease Type C (NPC) total market size, the market size by therapies, and market share (%) distribution in 2019, and how would it all look in 2032? What are the contributing factors for this growth?
- What will be the impact on the market with the approval of Arimoclomol?
- How much of the market will new therapies capture after their launch?
- What are the pricing variations among different geographies for Arimoclomol and AZ-3102?
- How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
Epidemiology Insights
What are the disease risks, burdens, and unmet needs of Niemann-Pick Disease Type C? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Niemann-Pick Disease Type C?- What is the historical and forecasted Niemann-Pick Disease Type C patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
- How many male and female patients have Niemann-Pick Disease Type C?
- How many Niemann-Pick Disease Type C patients are in the type groups of Early-infantile (< 2 years), Late-infantile (2 to < 6 years), juvenile (6 to < 15 years), and Adolescent/adult onset (=15 years)?
- What factors affect the diagnosis of Niemann-Pick Disease Type?
Current Treatment Scenario, Marketed Drug, and Emerging Therapies
- What are the current options for the standard of care for Niemann-Pick Disease Type C?
- What are the current guidelines for treating Niemann-Pick Disease Type C in the US and Europe?
- How many companies are developing therapies for treating Niemann-Pick Disease Type C?
- How many emerging therapies are in the mid-stage and late development stage for treating Niemann-Pick Disease Type C?
- What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
- What are the patents of emerging therapies for Niemann-Pick Disease Type C?
- What will impact the market after the expected patent expiry of the emerging drug?
- What is the cost burden of marketed therapies on patients?
- What will the patient acceptability regarding preferred treatment options as per real-world scenarios?
- What are the country-specific accessibility issues of recommended therapies?
- What are the 7MM historical and forecasted market of Niemann-Pick Disease Type C?
Reasons to Buy
- The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the Niemann-Pick Disease Type C Market.
- Insights on patient burden/disease prevalence, accuracy/evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
- To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
- Distribution of historical and current patient share based on real-world prescription data and reported sales of current products in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
- Identifying strong upcoming players in the market will help devise strategies that will help get ahead of competitors.
- Detailed analysis and potential of current and emerging therapies to provide visibility around leading emerging drugs.
- Highlights of Access and Reimbursement policies for MIGLUSTAT.
- To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
- Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
Table of Contents
1. Key Insights2. `Report Introduction4. Epidemiology and Market Forecast Methodology5. Key Events6. Executive Summary of Niemann-Pick Disease Type C9. Patient Journey12. KOL Views13. SWOT Analysis14. Unmet Needs17. Report Methodology18. Publisher Capabilities19. Disclaimer
3. Niemann-Pick Disease Type C Market Overview at a Glance
7. Disease Background and Overview: Niemann-Pick Disease Type C (NPC)
8. Epidemiology and Patient Population of Niemann-Pick Disease Type C
10. Emerging Therapies
11. Niemann-Pick Disease Type C: Seven Major Market Analysis
15. Market Access and Reimbursement
16. Appendix
List of Tables
List of Figures
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Zevra Therapeutics
- Azafaros A.G.
- Cyclo Therapeutics, Inc.
- IntraBio
