Dublin, May 06, 2024 (GLOBE NEWSWIRE) -- The "Niemann-Pick Disease Type C - Market Insight, Epidemiology And Market Forecast - 2032" report has been added to ResearchAndMarkets.com's offering.
The report delivers an in-depth understanding of the Niemann-Pick Disease Type C, historical and forecasted epidemiology as well as the Niemann-Pick Disease Type C market trends in the United States, EU4, the United Kingdom and Japan.
The 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.
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.
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) 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.
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
Featured Companies includes:
- Zevra Therapeutics
- Azafaros A.G.
- Cyclo Therapeutics, Inc.
- IntraBio
For more information about this report visit https://www.researchandmarkets.com/r/aufmpx
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