Niemann-Pick Disease Type C Market

• The Niemann-Pick Disease Type C Market Size was valued ~USD 34 million in 2022 and is anticipated to grow with a significant CAGR during the forecast period (2020-2034).
• In the 7MM, the United States accounted for the largest Niemann-Pick Disease Type C Market Size in 2023. With the expected launch of potential therapies, the Niemann-Pick Disease Type C Market Size is expected to increase during the forecast period (2024-2034).
• Among the Niemann-Pick disease types C1 and C2, type C1 accounts for more than 90% of the cases.
• In March 2024, Zevra Therapeutics announced that the United States Food and Drug Administration (US FDA) extended the review period for the New Drug Application (NDA) for arimoclomol, for the Niemann-Pick disease type C Treatment. The US FDA has set a new Prescription Drug User Fee Act (PDUFA) action date of September 21, 2024.
• In March 2024, the US FDA accepted IntraBio’s NDA and granted priority review for IB1001 for the treatment of Niemann-Pick disease type C. The application has been granted Priority Review and a PDUFA target action date of September 24, 2024.

 Request for Unlocking the Sample Page of the "Niemann-Pick Disease Type C Drugs Market"

DelveInsight’s "Niemann-Pick Disease Type C Drugs Market Insight, Epidemiology, and Market Forecast – 2034" report delivers an in-depth understanding of Niemann-Pick disease type C, historical and forecasted epidemiology as well as Niemann-Pick disease type C therapeutics market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan. 

Niemann-Pick disease type C Drugs Market Report provides current treatment practices, emerging drugs, Niemann-Pick disease type C market share of individual therapies, and current and forecasted Niemann-Pick disease type C market size from 2020 to 2034, segmented by seven major markets. The report also covers current Niemann-Pick disease type C treatment market practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the Niemann-Pick Disease Type C therapeutics market.

Niemann-Pick Disease Type C Treatment Market

Niemann-Pick disease type C is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. The accumulation of these substances damages the affected areas. Niemann-Pick disease type C is highly variable and the age of onset and specific symptoms can vary from one person to another, sometimes even among members of the same family. Niemann-Pick disease type C can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. Most cases are detected during childhood and progress to cause life-threatening complications by the second or third decade of life. Niemann-Pick disease type C is caused by mutations in the NPC1 gene (NPC type 1C) or the NPC2 gene (NPC type 2C) and is inherited in an autosomal recessive manner.

Niemann-Pick Disease Type C Diagnosis

Diagnosing Niemann-Pick disease typically involves a thorough physical examination, where signs such as enlarged liver or spleen may indicate the condition. Due to its rarity and symptom overlap with other illnesses, further testing is crucial for an accurate diagnosis. The specific type of Niemann-Pick disease dictates the diagnostic approach: for Type C, oxysterol levels are assessed. Genetic testing can reveal gene mutations associated with all types, identifying carriers and aiding in familial risk assessment. Imaging techniques like MRI help visualize organ abnormalities, especially in the brain, liver, and spleen. Additionally, eye examinations may detect disease-related changes, particularly in Type C. Prenatal testing through ultrasound or amniotic fluid/tissue sampling can diagnose Niemann-Pick disease in utero, facilitating early intervention and management.

Further details related to diagnosis will be provided in the report...

Niemann-Pick Disease Type C Treatment

Current Niemann-Pick Disease Type C Treatment is directed towards the specific symptoms apparent in each individual and is supportive and not curative. Regular monitoring and assessment of difficulty swallowing are essential to prevent the risk of aspiration. Initially, managing swallowing difficulties may involve softening solid foods and thickening liquids, with support from a speech therapist to enhance swallowing function. In cases where these interventions are insufficient, the placement of a gastrostomy tube may be necessary to ensure adequate calorie intake. This procedure involves inserting a thin tube directly into the stomach through a small incision in the abdomen, facilitating the direct delivery of food or medication.

Further details related to treatment will be provided in the report...

Niemann-Pick Disease Type C Epidemiology

The Niemann-Pick disease type C epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by 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, and Niemann-Pick Disease Type C treated cases in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.

• The United States accounted for the highest number of Niemann-Pick disease type C Prevalent Cases in the 7MM in 2023.
• Among gene-specific cases, Niemann-Pick disease type C1 accounted for most of the cases.
• Among the gender-specific cases, Niemann-Pick disease type C1 affects males and females equally.

Niemann-Pick Disease Type C Drugs Market Chapters

The drug chapter segment of the Niemann-Pick disease type C Drugs Market Report encloses a detailed analysis of the late-stage (Phase III ) and mid-stage (Phase II/III and Phase II) Niemann-Pick Disease Type C pipeline drugs analysis. The current key Niemann-Pick Disease Type C Companies such as Cyclo Therapeutics (Trappsol cyclo), IntraBio (IB10001), Zevra Therapeutics (Arimoclomol), and others. The drug chapter also helps understand the Niemann-Pick disease type C Clinical Trials details, pharmacological action, agreements and collaborations, approval, patent details, and the latest news and press releases.

Niemann-Pick Disease Type C Marketed Drugs

• ZAVESCA/BRAZAVES (miglustat): Actelion Pharmaceuticals

ZAVESCA/BRAZAVES is a small iminosugar molecule that reversibly inhibits glycosphingolipid synthesis and blocks the synthesis of glycosphingolipids, one of the substances that accumulate in the brain of individuals with Niemann-Pick disease type C and can slow the progression of neurological symptoms associated with Niemann-Pick disease type C. It was approved by the EMA in 2009. It has been denied approval for the treatment of Niemann-Pick disease type C, by the US FDA on the grounds of insufficient data. However, it is being prescribed to the Niemann-Pick disease type C patients as an off-label therapy.

Emerging Niemann-Pick Disease Type C Drugs

• Trappsol Cyclo: Cyclo Therapeutics

Trappsol by Cyclo Therapeutics is a proprietary formulation of hydroxypropyl betacyclodextrin and in multiple Niemann-Pick Disease Type C Clinical Trials, has shown encouraging results to effectively manage the transportation of cholesterol. Taking the place of the defective Niemann-Pick disease type C protein, Trappsol Cyclo, with its cyclic structure, captures cholesterol within the cell and transports it out of the cell. The drug is currently being evaluated in Phase III TransportNPC pivotal Niemann-Pick Disease Type Clinical Trials in pediatric and adult patients with Niemann-Pick disease type C1. It has received an Orphan drug designation by the US FDA and the EMA. It has also received Rare Pediatric Disease Designation, and Fast-track designation by the US FDA for the treatment of Niemann-Pick disease type C. Recently, the company announced a positive outcome from its recent Type C meeting with the US FDA to discuss the review of the Trappsol Cyclo clinical development program to date, and proposed strategies for the statistical analysis for the ongoing pivotal Phase III study, TransportNPC. With this aligned strategy, interim analysis data readout is anticipated for the first quarter of 2025.

• IB10001: IntraBio

IntraBio’s lead drug series, IB1000s, is a set of orally administered, modified amino acids, N-acetyl-leucine, categorized by a well-established safety and tolerability profile. IB1001 (N-acetyl-l-leucine) is being developed for Niemann-Pick Disease Type C, besides other orphan indications like Tay-Sachs and Sandhoff Disease, and inherited Cerebellar Ataxias. IB1000s have an effect on the normalization of neuronal membrane potential and intracellular ion regulation via calcium channels and have been observed to have symptomatic and neuroprotective properties and disease-modifying potential. The US FDA has accepted IntraBio’s NDA and granted priority review for IB1001 for the Niemann-Pick Disease Type C Treatment. The application has been granted Priority Review and a PDUFA target action date of September 24, 2024.

Niemann-Pick Disease Type C Market Outlook

Various supportive therapies are available for managing symptoms linked with Niemann-Pick disease type C, including antiepileptics for seizures, tricyclic antidepressants, CNS stimulants for cataplexy, and anticholinergics for dystonia and tremor. However, these Niemann-Pick Disease Type C Therapies contribute minimally to market revenue for Niemann-Pick disease type C1 treatment. ZAVESCA, prescribed to approximately half of Niemann-Pick disease type C patients, offers hope amidst the lack of disease-targeting therapies. Challenges in Niemann-Pick disease type C market growth include difficulties in diagnosing the condition due to its association with other symptoms and the limited number of approved drugs necessitating a wider therapeutic index. Extensive research is needed for both treatment and diagnostics. While bone marrow or liver transplantation may alleviate certain symptoms, they do not effectively address neurological issues in Niemann-Pick disease type C1 patients. Encouragingly, early hematopoietic stem cell transplantation shows promise for Niemann-Pick disease type C2 patients.

Key Findings

• In the 7MM, the United States accounts for the largest Niemann-Pick Disease Type C Market Size.
• The Niemann-Pick Disease Type C Market Size is expected to increase during the forecast period with the increasing prevalence and launch of new therapies such as IB10001 and Trappsol Cyclo shall fuel the growth of the market.

Niemann-Pick Disease Type C Drugs Uptake

This section focuses on the uptake rate of potential Niemann-Pick Disease Type C drugs expected to be launched in the market during 2024–2034. The Niemann-Pick Disease Type C treatment market landscape has experienced a transformation with the uptake of novel drugs. These innovative therapies are redefining standards of care. Furthermore, the increased uptake of these transformative drugs is a testament to the unwavering dedication of Neurologists, professors of neurology, gastroenterologists, and professors in the Department of Translational Neuroscience. This momentous shift in treatment paradigms is a testament to the power of research, collaboration, and human resilience.

Niemann-Pick disease type C Pipeline Development Activities

The Niemann-Pick Disease Type C drugs market report provides insights into Niemann-Pick Disease Type C clinical trials within Phase III, Phase II/III, and Phase II. It also analyzes key Niemann-Pick Disease Type C Companies involved in developing targeted therapeutics. Niemann-Pick Disease Type C Companies like Cyclo Therapeutics, IntraBio, and others actively engage in late and mid-stage research and development efforts for Niemann-Pick disease type C. There is a positive outlook for the Niemann-Pick Disease Type C Therapeutics Market, with expectations of growth during the forecast period (2024–2034).

Pipeline Development Activities

The Niemann-Pick Disease Type C drugs market report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Niemann-Pick disease type C.

KOL Views

To keep up with current Niemann-Pick Disease Type C Treatment 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 the evolving Niemann-Pick Disease Type C Treatment Market landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Neurology specialists, Neuroscience specialists, and others. 

DelveInsight’s analysts connected with 15+ KOLs to gather insights; however, interviews were conducted with 5+ KOLs in the 7MM. Centers such as the University of Nebraska Medical Center, Centers for Disease Control and Prevention, Department of Translational Neuroscience, etc., were contacted. Their opinion helps understand and validate current and emerging therapy 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

Niemann-Pick Disease Type C Drugs Market: Qualitative Analysis

We perform Qualitative and Niemann-Pick Disease Type C Treatment Market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of Niemann-Pick Disease Type C 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 Niemann-Pick Disease Type C treatment market landscape.

Niemann-Pick Disease Type C Therapeutics Market Access and Reimbursement

Reimbursement is the price negotiation between the manufacturer and payer that allows the manufacturer access to the Niemann-Pick Disease Type C Therapeutics Market. It is provided to reduce the high costs and make essential Niemann-Pick Disease Type C Drugs affordable. In the US healthcare system, both Public and Private health insurance coverage are included. In addition, Medicare and Medicaid are the largest government-funded programs in the US. The major healthcare programs, including Medicare, Medicaid, the Children’s Health Insurance Program (CHIP), and the state and federal health insurance marketplaces, are overseen by the Centers for Medicare & Medicaid Services (CMS). Other than these, Pharmacy Benefit Managers (PBMs), and third-party organizations that provide services and educational programs to aid patients are also present. The Niemann-Pick Disease Type C Drugs Therapeutics Market Report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of currently used 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.

Niemann-Pick Disease Type C Therapeutics Market Report Scope

• The Niemann-Pick Disease Type C therapeutics market report covers a segment of key events, an executive summary, and a descriptive overview, explaining its causes, signs, symptoms, pathogenesis, and currently used therapies.
• Comprehensive insight into the Niemann-Pick Disease Type C epidemiology segments and forecasts, disease progression, and Niemann-Pick Disease Type C treatment market guidelines has been provided.
• Additionally, an all-inclusive account of the emerging therapies and the elaborate profiles of late-stage and prominent therapies will impact the current Niemann-Pick Disease Type C Treatment Market landscape.
• A detailed review of the Niemann-Pick disease type C treatment market, historical and forecasted Niemann-Pick Disease Type C market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The Niemann-Pick Disease Type C treatment market 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 Niemann-Pick disease type C.

Niemann-Pick Disease Type C Therapeutics Market Report Insights

• Patient-based Niemann-Pick Disease Type C Market Forecasting
• Niemann-Pick Disease Type C Therapeutic Approaches
• Niemann-Pick Disease Type C Pipeline Drugs Analysis
• Niemann-Pick Disease Type C Treatment Market Size and Trends
• Existing and Future Niemann-Pick Disease Type C Therapeutics Market Opportunity

Niemann-Pick Disease Type C Therapeutics Market Report Key Strengths

• 11 Years Niemann-Pick Disease Type C Market Forecast
• The 7MM Coverage
• Niemann-Pick Disease Type C Epidemiology Segmentation
• Key Cross Competition
• Niemann-Pick Disease Type C Drugs Uptake
• Key Niemann-Pick Disease Type C Market Forecast Assumptions

Niemann-Pick Disease Type C Treatment Market Report Assessment

• Current Niemann-Pick Disease Type C Treatment Market Practices
• Niemann-Pick Disease Type C Unmet Needs
• Niemann-Pick Disease Type C Pipeline Drugs Profiles
• Niemann-Pick Disease Type C Therapeutics Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)
• Niemann-Pick Disease Type C Market Drivers
• Niemann-Pick Disease Type C Market Barriers

FAQs

• What was the Niemann-Pick disease type C Treatment Market Size, the Niemann-Pick Disease Type C Market Size by therapies, Niemann-Pick Disease Type C market share (%) distribution in 2023, and what would it look like by 2034? What are the contributing factors for this growth?
• What can be the future treatment paradigm for Niemann-Pick disease type C?
• What are the disease risks, burdens, and Niemann-Pick disease type C unmet needs ? What will be the growth opportunities across the 7MM concerning the patient population with Niemann-Pick disease type C?
• What are the current options for the Niemann-Pick disease type C treatment? What are the current guidelines for treating Niemann-Pick disease type C in the 7MM?
• What are the recent novel therapies, targets, Niemann-Pick Disease Type C Mechanisms of Action, and technologies being developed to overcome the limitations of existing therapies?
• What is the patient's share in Niemann-Pick disease type C?

Reasons to Buy

• The Niemann-Pick Disease Type C treatment market report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving Niemann-Pick disease type C
• Insights on patient burden/disease Niemann-Pick Disease Type C prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing Niemann-Pick Disease Type C treatment market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming Niemann-Pick Disease Type C Companies in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis ranking of class-wise potential current and emerging therapies to provide visibility around leading classes.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label Niemann-Pick Disease Type C Therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives 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 Niemann-Pick Disease Type C therapeutics market so that the upcoming Niemann-Pick Disease Type C Companies can strengthen their development and launch strategy.

Access Exclusive Data Now! Click here to Read More about the Related Articles:-

• Emerging Therapies for Niemann-Pick Disease Type C Treatment: The Road to Progress
• Niemann–Pick disease type C Therapeutics Market
• Niemann-Pick disease type C (NPC) -A Rare Cognitive Decline Indication
• Latest DelveInsight Blogs