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ニーマンピック病C型(NPC)- 市場洞察、疫学、市場予測 2028年

Niemann-Pick Disease Type C (NPC) - Market Insights, Epidemiology, and Market Forecast-2028

発行 DelveInsight Business Research LLP 商品コード 650463
出版日 ページ情報 英文 166 Pages
納期: 即日から翌営業日
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ニーマンピック病C型(NPC)- 市場洞察、疫学、市場予測 2028年 Niemann-Pick Disease Type C (NPC) - Market Insights, Epidemiology, and Market Forecast-2028
出版日: 2019年07月01日 ページ情報: 英文 166 Pages
概要

主要7カ国(米国、英国、ドイツ、フランス、イタリア、スペイン、日本)のニーマンピック病C型(NPC)の有病数は2017年に2,116人と推計されています。

当レポートでは、主要7カ国(米国、英国、ドイツ、フランス、イタリア、スペイン、日本)におけるニーマンピック病C型(NPC)市場を調査し、疾病の概要や現行の治療法、新薬のプロファイル、全体および各国の市場動向、疫学的予測、市場規模、有病数・診断数の推移と予測、アンメットニーズ、市場の成長要因および障壁などを包括的にまとめています。

目次

第1章 主な洞察

第2章 市場概要

  • 市場シェア(実績値)
  • 市場シェア(予測値)

第3章 疾患背景と概要

  • イントロダクション
  • 疾病の分類
  • 原因
  • 兆候と症状
    • 発症年齢別
  • 遺伝パターン
  • 病態生理学
  • 診断

第4章 疫学と患者人口

  • 主な調査結果
  • 主要7カ国のニーマンピック病C型の総有病数
  • 主要7カ国のニーマンピック病C型の総診断数

第5章 ニーマンピック病C型の疫学:国別

  • 米国
    • 総有病数
    • 総有病数:病型別
    • 診断数
    • 診断数:臨床型別
  • EU5カ国
    • ドイツ
      • 総有病数
      • 総有病数:病型別
      • 診断数
    • フランス
      • 総有病数
      • 総有病数:病型別
      • 診断数
    • イタリア
      • 総有病数
      • 総有病数:病型別
      • 診断数
    • スペイン
      • 総有病数
      • 総有病数:病型別
      • 診断数
    • 英国
      • 総有病数
      • 総有病数:病型別
      • 診断数
  • 日本
    • 総有病数
    • 総有病数:病型別
    • 診断数

第6章 治療の実践

  • 治療目標
  • 治療アルゴリズム
  • ニーマンピック病C型の臨床管理ガイドライン

第7章 アンメットニーズ

第8章 上市済み治療薬

  • ザベスカ:Actelion Pharmaceuticals
    • 薬の説明
    • 規制のマイルストーン
    • 後発医薬品の入手性
    • 臨床開発
    • 安全性と有効性
    • 製品プロファイル

第9章 新薬

  • キークロス分析
  • アリモクロモール:Orphazyme
    • 医薬品概要
    • 開発活動
    • 臨床開発
    • 安全性と有効性
    • 製品プロファイル
  • VTS-270:Vtesse(マリンクロット)
  • Trappsol Cyclo(シクロデキストリン):CTD Holdings
  • IB1001:IntraBio

第10章 主要7カ国市場の分析

  • 主な調査結果
  • 主要7カ国の総市場規模
  • 主要7カ国の市場規模:薬効分類別
  • 主要7カ国の市場規模:治療薬別

第11章 米国市場の見通し

  • 総市場規模
  • 市場規模:薬効分類別
  • 市場規模:治療薬別

第12章 EU5カ国市場の見通し

  • ドイツ
    • 総市場規模
    • 市場規模:薬効分類別
    • 市場規模:治療薬別
  • フランス
  • イタリア
  • スペイン
  • 英国

第13章 日本市場の見通し

  • 総市場規模
  • 市場規模:薬効分類別
  • 市場規模:治療薬別

第14章 市場の成長要因

第15章 市場の障壁

第16章 付録

  • 調査手法

第17章 DelveInsightのサービス内容

第18章 免責事項

第19章 DelveInsightについて

図表

List of Tables

  • Table 1: Types of Neiman-Pick Disease type, distinguish features, primary pathophysiology, and genetics issue.
  • Table 2: Sign and symptom combination strongly suggestive of NPC
  • Table 3: Cell biology diagnostic techniques in NPC
  • Table 4: Primary and urinary diagnostic biomarkers in NPC
  • Table 5: Clinical patient groups with an increased risk of Niemann-Pick Disease Type C
  • Table 6: Diagnosis
  • Table 7: Recommendations for diagnosis of patients identified by molecular genetic screening for NPC1 and NPC2
  • Table 8: NPC suspicion index tool: Signs and symptoms
  • Table 9: Total Prevalent Patient Population of NPC in 7MM (2017-2028)
  • Table 10: Total Diagnosed Prevalent Patient Population of NPC in the 7MM (2017-2028)
  • Table 11: Total Prevalent Population of NPC in the United States (2017-2028)
  • Table 12: Sub-type Specific Prevalence of NPC in the United States (2017-2028)
  • Table 13: Diagnosed Population of NPC in the United States (2017-2028)
  • Table 14: Diagnosed Prevalence of NPC based on Clinical Phenotypesin the United States (2017-2028)
  • Table 15: Total Prevalent Population of NPC in Germany (2017-2028)
  • Table 16: Sub-type Specific Prevalence of NPC in Germany (2017-2028)
  • Table 17: Diagnosed Population of NPC in the United States (2017-2028)
  • Table 18: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Germany (2017-2028)
  • Table 19: Total Prevalent Population of NPC in France (2017-2028)
  • Table 20: Sub-type Specific Prevalence of NPC in France (2017-2028)
  • Table 21: Diagnosed Population of NPC in France (2017-2028)
  • Table 22: Diagnosed Prevalence of NPC based on Clinical Phenotypes in France (2017-2028)
  • Table 23: Total Prevalent Population of NPC in Italy (2017-2028)
  • Table 24: Sub-type Specific Prevalence of NPC in Italy (2017-2028)
  • Table 25: Diagnosed Population of NPC in Italy (2017-2028)
  • Table 26: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Italy (2017-2028)
  • Table 27: Total Prevalent Population of NPC in Spain (2017-2028)
  • Table 28: Sub-type Specific Prevalence of NPC in Spain (2017-2028)
  • Table 29: Diagnosed Population of NPC in Spain (2017-2028)
  • Table 30: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Spain (2017-2028)
  • Table 31: Total Prevalent Population of NPC in the UK (2017-2028)
  • Table 32: Sub-type Specific Prevalence of NPC in the UK (2017-2028)
  • Table 33: Diagnosed Population of NPC in the United Kingdom (2017-2028)
  • Table 34: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the UK (2017-2028)
  • Table 35: Total Prevalent Population of NPC in Japan (2017-2028)
  • Table 36: Sub-type Specific Prevalence of NPC in Japan (2017-2028)
  • Table 37: Diagnosed Population of NPC in Japan (2017-2028)
  • Table 38: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Japan (2017-2028)
  • Table 39: Symptomatic Therapies for Niemann-Pick Disease type C
  • Table 40: Zavesca, Clinical Trial Description, 2019
  • Table 41: Emerging Drugs Analysis
  • Table 42: Arimoclomol, Clinical Trial Description, 2019
  • Table 43: VTS-270, Clinical Trial Description, 2019
  • Table 44: Trappsol Cyclo, Clinical Trial Description, 2019
  • Table 45: IB1001, Clinical Trial Description, 2019
  • Table 46: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2017-2028)
  • Table 47: 7MM Market Size of NPC by Therapeutic Class in USD Million (2017-2028)

Table 48 Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2017-2028)

  • Table 49: The US Market Niemann Pick Disease Type C in USD Million (2017-2028)
  • Table 50: The U.S. NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 51: The US NPC market size by Therapies in USD Million (2017-2028)
  • Table 52: Market Size of NPC in Germany, in USD Million (2017-2028)
  • Table 53: Germany NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 54: Germany NPC market size by Therapies in USD Million (2017-2028)
  • Table 55: Market Size of Niemann Pick Disease Type C in France, in USD Million (2017-2028)
  • Table 56: France NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 57: France NPC market size by Therapies in USD Million (2017-2028)
  • Table 58: Market Size of Niemann Pick Disease Type C in Italy, in USD Million (2017-2028)
  • Table 59: Italy NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 60: Italy NPC market size by Therapies in USD Million (2017-2028)
  • Table 61: Market Size of Niemann Pick Disease Type C in Spain, in USD Million (2017-2028)
  • Table 62: Spain NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 63: Spain NPC market size by Therapies in USD Million (2017-2028)
  • Table 64: Market Size of Niemann Pick Disease Type C in the UK, in USD Million (2017-2028)
  • Table 65: The UK NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Table 66: Emerging Therapies market of Niemann Pick Disease Type C in the UK, in USD Million (2017-2028)
  • Table 67: Market Size of Niemann Pick Disease Type C in Japan, in USD Million (2017-2028)
  • Table 68: Japan Niemann Pick Disease Type C Market by Therapeutic Class in USD Million (2017-2028)
  • Table 69: Japan NPC market size by Therapies in USD Million (2017-2028)

List of Figures

  • Figure 1: Classification of Niemann Pick Disease
  • Figure 2: Classification of Niemann-Pick Disease Type C
  • Figure 3: Classification of signs and symptoms in NPC
  • Figure 4: Manifestations of Niemann-Pick Disease Type C (NPC) by age group
  • Figure 5: Schematic representation of Niemann-Pick Disease Type C
  • Figure 6: Inheritance Pattern of Niemann-Pick Disease Type C (NPC)
  • Figure 7: Pathophysiology of Niemann-Pick Disease Type C 1 Disease
  • Figure 8: Pathophysiology of Niemann-Pick Disease Type C 2 Disease
  • Figure 9: Diagnostic Algorithm for NPC
  • Figure 10: Diagnostic Algorithm for Niemann-Pick Disease Type C
  • Figure 11: Total Prevalent Patient Population of NPC in 7MM (2017-2028)
  • Figure 12: Total Diagnosed Prevalent Patient Population of NPC in the 7MM (2017-2028)
  • Figure 13: Prevalence of NPC in the United States (2017-2028)
  • Figure 14: Sub-type Specific Prevalence of NPC in the United States (2017-2028)
  • Figure 15: Diagnosed Population of NPC in the United States (2017-2028)
  • Figure 16: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the United States (2017-2028)
  • Figure 17: Total Prevalent Population of NPC in Germany (2017-2028)
  • Figure 18: Sub-type Specific Prevalence of NPC in Germany (2017-2028)
  • Figure 19: Diagnosed Population of NPC in Germany (2017-2028)
  • Figure 20: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Germany (2017-2028)
  • Figure 21: Total Prevalent Population of NPC in France (2017-2028)
  • Figure 22: Sub-type Specific Prevalence of NPC in France (2017-2028)
  • Figure 23: Diagnosed Population of NPC in France (2017-2028)
  • Figure 24: Diagnosed Prevalence of NPC based on Clinical Phenotypes in France (2017-2028)
  • Figure 25: Total Prevalent Population of NPC in Italy (2017-2028)
  • Figure 26: Sub-type Specific Prevalence of NPC in Italy (2017-2028)
  • Figure 27: Diagnosed Population of NPC in Italy (2017-2028)
  • Figure 28: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Italy (2017-2028)
  • Figure 29: Total Prevalent Population of NPC in Spain (2017-2028)
  • Figure 30: Sub-type Specific Prevalence of NPC in Spain (2017-2028)
  • Figure 31: Diagnosed Population of NPC in Spain (2017-2028)
  • Figure 32: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Spain (2017-2028)
  • Figure 33: Total Prevalent Population of NPC in the UK (2017-2028)
  • Figure 34: Sub-type Specific Prevalence of NPC in the UK (2017-2028)
  • Figure 35: Diagnosed Population of NPC in the United Kingdom (2017-2028)
  • Figure 36: Diagnosed Prevalence of NPC based on Clinical Phenotypes in the UK (2017-2028)
  • Figure 37: Total Prevalent Population of NPC in Japan (2017-2028)
  • Figure 38: Sub-type Specific Prevalence of NPC in Japan (2017-2028)
  • Figure 39: Diagnosed Population of NPC in Japan (2017-2028)
  • Figure 40: Diagnosed Prevalence of NPC based on Clinical Phenotypes in Japan (2017-2028)
  • Figure 41: Treatment Algorithm for Niemann-Pick Disease Type C (NPC)
  • Figure 42: Unmet Needs for Niemann-Pick Disease Type C
  • Figure 43: 7 Major Market Size of Niemann Pick Disease Type C in USD Million (2017-2028)
  • Figure 44: 7MM Market Size of NPC by Therapeutic Class in USD Million (2017-2028)
  • Figure 45: Market size of Niemann Pick Disease Type C by Therapies in 7MM, USD Million (2017-2028)
  • Figure 46: Market Size of NPC in the United States, USD Millions (2017-2028)
  • Figure 47: The U.S. NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 48: The U.S. market size of NPC by therapies in USD Million (2017-2028)
  • Figure 49: Market Size of NPC in Germany, USD Millions (2017-2028)
  • Figure 50: Germany NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 51: Germany NPC market size by Therapies in USD Million (2017-2028)
  • Figure 52: Market Size of Niemann Pick Disease Type C in France, USD Millions (2017-2028)
  • Figure 53: France NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 54: France NPC market size by Therapies in USD Million (2017-2028)
  • Figure 55: Market Size of Niemann Pick Disease Type C in Italy, USD Millions (2017-2028)
  • Figure 56: Italy NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 57: Italy NPC market size by Therapies in USD Million (2017-2028)
  • Figure 58: Market Size of Niemann Pick Disease Type C in Spain, USD Millions (2017-2028)
  • Figure 59: Spain NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 60: Spain NPC market size by Therapies in USD Million (2017-2028)
  • Figure 61: Market Size of Niemann Pick Disease Type C in the UK, USD Millions (2017-2028)
  • Figure 62: The UK NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 63: The UK NPC market size by Therapies in USD Million (2017-2028)
  • Figure 64: Market Size of Niemann Pick Disease Type C in Japan, USD Millions (2017-2028)
  • Figure 65: Japan NPC Market by Therapeutic Class in USD Million (2017-2028)
  • Figure 66: Japan NPC market size by Therapies in USD Million (2017-2028)
  • Figure 67: Market Drivers for Niemann-Pick Disease Type C
  • Figure 68: Market Barriers for Niemann-Pick Type C Disease
目次
Product Code: DIMI0350

DelveInsight's 'Niemann-Pick Disease Type C (NPC)- Market Insights, Epidemiology, and Market Forecast-2028 report delivers an in-depth understanding of the disease, historical, and forecasted epidemiology as well as the market trends of NPC in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

The report provides the current treatment practices, emerging drugs, market share of the individual therapies, the current and forecasted market size of NPC from 2017 to 2028 segmented by the seven major markets. The report also covers current treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate best of the opportunities and assess the underlying potential of the market.

Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2017-2028

Niemann-Pick Disease Type C (NPC) - Disease Understanding and Treatment Algorithm

Niemann-Pick disease type C (NPC) 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 inability leads to the abnormal accumulation of these cholesterol and other fatty substances within various tissues of the body and brain tissue.

NPC varies according to the different age of onset and symptoms can vary from one person to another person. This genetic disorder characterized by the visceral, neurological, and psychiatric manifestations. At the cellular level, NPC can be caused by a mutation in the NPC1 gene or NPC2 gene and is inherited in an autosomal recessive manner. NPC has an extremely heterogeneous clinical presentation, and there is considerable variation when type C symptoms first appear (enlarged liver, enlarged spleen, or jaundice) and the progression of the disease.

The diagnosis of NPC is completely based on the characteristic symptoms like psychiatric issues, neurologic findings, etc. Sometimes this disorder is misdiagnosed for other associated neurological disorder.

According to the National Organization of Rare Disorders (NORD), NPC equally affects males and females.

DelveInsight's NPC market report gives a thorough understanding of the NPC by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for NPC in the US, Europe, and Japan.

Niemann-Pick Disease Type C (NPC) - Epidemiology

The NPC epidemiology division provides insights about historic and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight's report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology [segmented by Total Prevalent Population, Subtype-Specific prevalent population, Diagnosed Prevalent Population, and prevalence of diagnosed cases of NPC based on Clinical Phenotypes] scenario of NPC in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom) and Japan from 2017 to 2028.

According to DelveInsight, total prevalent population of Niemann-Pick disease type C (NPC) in the 7MM was observed to be 2,116 in 2017. DelveInsight's epidemiology model for NPC estimates that NPC1 subtype is the most prevalent in the 7MM countries, as compared to NPC2 subtype. DelveInsight's estimate suggests the highest prevalence of NPC in the United States with 1,011 cases in 2017.

Furthermore, among the European 5 countries, Germany had the highest diagnosed prevalent population of NPC with 251 cases in 2017, followed by France and the United Kingdom. On the other hand, Spain had the lowest diagnosed prevalent population of 151 in 2017. Moreover, Japan witnessed 102 diagnosed prevalent cases for NPC in 2017. According to DelveInsight's analysts, the total diagnosed prevalent population of NPC in the 7MM was assessed to be 1,058 in 2017.

According to DelveInsight's epidemiology model for NPC, based on phenotypes of the disease, suggests that of the total NPC cases in the 7MM, varied significantly among different countries. In the United States, juvenile cases constituted the majority of the patient population, whereas, in the United Kingdom, most commonly occurring was observed to be Early infantile.

Niemann-Pick disease type C (NPC) - Drug Chapters

This segment of the NPC report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the 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.

Zavesca (Actelion Pharmaceuticals) is the only approved therapy for the treatment of progressive neurological manifestations in adult patients and pediatric patients with NPC disease in Europe and Japan. However, this drug is not approved for NPC in the United States; hence, it is being used as an off-label therapy for the treatment of NPC in the United States. The approval has been denied on has been denied by the US FDA on the ground of insufficient data. FDA requested for more data, which would take years to get, and almost impossible to satisfy the high standards of the FDA, mainly restrained by the limited or lack of patient population which could be recruited in the trials. However, since the drug is already approved for the treatment of Gaucher's disease in the US, thus it is being prescribed to the NPC patients as an off-label therapy.

VTS-270 (Mallinckrodt) also known as Kleptose; 2-Hydroxypropyl- beta-cyclodextrin; HP-beta- CD; HPBCD) is under development. The drug has received various designations from the United States Food Drug and Administration (FDA)-Rare Pediatric Disease Designation, Promising Innovative Medicine (PIM) Designation, Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of NPC. VTS-270 is an intrathecal preparation that can cross the Blood Brain Barrier (BBB) and as a result, it targets the neurological symptoms of NPC such as dementia and cataplexy.

Trappsol Cyclo (CTD Holdings), the drug is under Phase-I/IIa stage of development for treating NPC. It has also received Rare Pediatric Disease Designation, Orphan Drug Designation and Fast-track designation by the US FDA for the treatment of NPC. Trappsol Cyclo is administered via intravenous route and thus targets the visceral symptoms, affecting the peripheral organs such as the spleen, liver, and lungs.

Both VTS-270 and Trappsol Cyclo drugs have already been given to the NPC patients under the Compassionate use Program.

Arimoclomol (Orphazyme) is a chaperone therapy proved as a great therapeutic option for the management of Lysosomal storage disorders [LSDs]. It is a small molecule activator of chaperones present in cells under stress, which is under development for the treatment of Niemann-Pick Disease and Gaucher's Disease. The drug candidate is under Phase II/III stage of development for the treatment of NPC. It is an orally administered drug with the bioavailability of 80-90% that acts by targeting heat shock protein 70 (HSP 70). The US FDA has granted Fast Track Designation for the Phase III clinical study of Arimoclomol for the treatment of NPC.

Niemann-Pick disease type C (NPC) - Market Outlook

The NPC market outlook of the report helps to build the detailed comprehension of the historical, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a thorough detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market, and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

At present, the therapeutic market size of NPC in the United States is mainly accounted by Substrate Reduction Therapy i.e., Zavesca [Miglustat; Actelion] and other supportive therapies [as per the recommendations of NP-C guidelines working group].

There are other various supportive therapies available for the management of symptoms associated with NPC. These therapies mainly include Antiepileptics (Seizures); Tricyclic Antidepressants & CNS Stimulants (Cataplexy); Anticholinergics, Trihexyphenydil & GABA Derivatives (Dystonia & Tremor); atypical antipsychotics (Psychosis). They account for a minimal towards the market revenue of NPC.

The therapeutic market of Niemann-Pick Type C (NPC) in the seven major markets was assessed to be USD 28.676 million in 2017 and is expected to grow during the study period (2017-2028). As per the assessments by DelveInsight's analysts, the market share of prescription therapies was found to be USD 9.254 million in 2017, in the United States. This was entirely dependent on supportive treatment regimens. Of the supportive treatment regimens, Zavesca, which has already been approved in the EU-5 countries and Japan, contributed USD 9.214 million in 2017 in the United States.

In contrast to the US therapeutic market, the current therapeutic market size of NPC in the EU-5 countries and Japan is dominated with the use of only approved drug Zavesca/Brazaves [Miglustat; Actelion] and the supportive therapies.

Advancement in the development of novel drug candidate by various pharmaceutical companies the dynamics of NPC market is anticipated to change in the coming years owing to the expected launch of emerging therapies during the forecasted period of 2019-2028. There are presently three emerging therapies focused on the treatment of NPC, which are expected to enter the market during the forecast period [2019-2028], namely VTS-270 (Mallinckrodt), Arimoclomol (Orphazyme), and Trappsol Cyclo (CTD Holdings, Inc.).

Niemann-Pick Disease Type C (NPC) - Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will launch in the market during the study period from 2017 to 2028. The analysis covers market uptake by drugs; patient uptake by therapies, and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

The dynamics of Niemann-Pick Disease Type C (NPC) market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the diseases, and advancement in the research and development of emerging therapies during the forecast period of 2019-2028. Companies across the globe are thoroughly working toward the development of new treatment therapies for NPC.

Key players like Mallinckrodt (VTS-270), CTD Holdings (Trappsol Cyclo), Orphazyme (Arimoclomol) and others are working towards the development of the eligible candidate for the treatment of NPC. While some of these are in the early stage of development.

Of the emerging therapies, VTS-270 (Mallinckrodt), Trappsol Cyclo (CTD Holdings), and Arimoclomol (Orphazyme) are expected to enter the treatment market, with their respective products, during the forecast period [2019-2028].

According to expert opinions, Zavesca is being prescribed to around half of the patients suffering from NPC. Miglustat has been a ray of hope amidst the lack of disease-targeting therapies, for years. However, since it's an off-label therapy, the recent US FDA approval of its generic version developed by Amerigen & Dipharma [in April 2018, for the treatment of Gaucher's disease], may impact the revenue generated by the drug, along with the launch of emerging therapies expected to launch in coming years.

Before the approval of Zavesca, only symptomatic therapies were used for the treatment of neurological manifestations, psychiatric manifestations and visceral manifestations associated with NPC. But advancement in the development of novel drug candidate by various pharmaceutical companies the dynamics of NPC market is anticipated to change in the coming years owing to the expected launch of emerging therapies during the forecasted period of 2019-2028. There are presently three emerging therapies focused on the treatment of NPC, which are expected to enter the market during the forecast period [2019-2028], namely VTS-270 (Mallinckrodt), Arimoclomol (Orphazyme), and Trappsol Cyclo (CTD Holdings, Inc.).

NPC Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

NPC Report Key Strengths

  • 10 Years Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Key Cross Competition
  • Market Size by Therapies
  • Drugs Uptake

NPC Report Assessment

  • Pipeline Product Profiles
  • Key Products and Key Players
  • Market Drivers and Barriers

Key Benefits

  • This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving the NPC market
  • Organize sales and marketing efforts by identifying the best opportunities for the NPC market
  • To understand the future market competition in the NPC market.

Table of Contents

1. Key Insights

2. Niemann-Pick Disease Type C (NPC) Market Overview at a Glance

  • 2.1. Market Share (%) Distribution of Niemann-Pick Disease Type C (NPC) in 2017
  • 2.2. Market Share (%) Distribution of Niemann-Pick Disease Type C (NPC) in 2028

3. Disease Background and Overview: Niemann-Pick Disease Type C (NPC)

  • 3.1. Introduction
  • 3.2. Classification of Niemann Pick Disease
  • 3.3. Causes
  • 3.4. Signs and Symptoms
    • 3.4.1. Sign and symptoms on the basis of the age of onset
  • 3.5. Inheritance patterns
  • 3.6. Pathophysiology
  • 3.7. Diagnosis
    • 3.7.1. NPC Diagnostic Recommendations Expert Panel: Diagnostic Recommendations (2016)
    • 3.7.2. NPC suspicion Index Tool

4. Epidemiology and Patient Population

  • 4.1. Key Findings
  • 4.2. 7MM Total Prevalent Patient Population of Niemann Pick Disease Type-C
  • 4.3. 7MM Total Diagnosed Prevalent Patient Population of Niemann Pick Disease Type-C

5. Country Wise-Epidemiology of NPC

  • 5.1. United States
    • 5.1.1. KOL Insights
    • 5.1.2. Total Prevalent Patient Population of NPC in the United States
    • 5.1.3. Sub-type Specific Prevalence of NPC in the United States
    • 5.1.4. Diagnosed Population of NPC in the United States
    • 5.1.5. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.2. EU5 Countries
    • 5.2.1. KOL Insights
  • 5.3. Germany
    • 5.3.1. Total Prevalent Patient Population of NPC in Germany
    • 5.3.2. Sub-type Specific Prevalence of NPC in Germany
    • 5.3.3. Diagnosed Population of NPC in Germany
    • 5.3.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.4. France
    • 5.4.1. Total Prevalent Patient Population of NPC in France
    • 5.4.2. Sub-type Specific Prevalence of NPC in France
    • 5.4.3. Diagnosed Population of NPC in France
    • 5.4.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.5. Italy
    • 5.5.1. Total Prevalent Patient Population of NPC in Italy
    • 5.5.2. Sub-type Specific Prevalence of NPC in Italy
    • 5.5.3. Diagnosed Population of NPC in Italy
    • 5.5.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.6. Spain
    • 5.6.1. Total Prevalent Patient Population of NPC in Spain
    • 5.6.2. Sub-type Specific Prevalence of NPC in Spain
    • 5.6.3. Diagnosed Population of NPC in Spain
    • 5.6.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.7. United Kingdom
    • 5.7.1. Total Prevalent Patient Population of NPC in the UK
    • 5.7.2. Sub-type Specific Prevalence of NPC in the UK
    • 5.7.3. Diagnosed Population of NPC in the United Kingdom
    • 5.7.4. Diagnosed Prevalence of NPC based on Clinical Phenotypes
  • 5.8. Japan
    • 5.8.1. KOL Insights
    • 5.8.2. Total Prevalent Patient Population of NPC in Japan
    • 5.8.3. Sub-type Specific Prevalence of NPC in Japan
    • 5.8.4. Diagnosed Population of NPC in Japan
    • 5.8.5. Diagnosed Prevalence of NPC based on Clinical Phenotypes

6. Treatment Practices

  • 6.1. Treatment Goals
  • 6.2. Treatment Algorithm
  • 6.3. Consensus clinical management guidelines for Niemann-Pick disease type C

7. Unmet Needs

8. Marketed drugs

  • 8.1. Zavesca: Actelion Pharmaceuticals
    • 8.1.1. Drug description
    • 8.1.2. Regulatory milestones
    • 8.1.3. Generic Availability
    • 8.1.4. Clinical Development
    • 8.1.5. Safety and efficacy
    • 8.1.6. Product Profile

9. Emerging drugs

  • 9.1. Key Cross Analysis
  • 9.2. Arimoclomol: Orphazyme
    • 9.2.1. Drug Description
    • 9.2.2. Other Developmental Activities
    • 9.2.3. Clinical Development
    • 9.2.4. Safety and Efficacy
    • 9.2.5. Product Profile
  • 9.3. VTS-270: Vtesse (Mallinckrodt)
    • 9.3.1. Drug Description
    • 9.3.2. Other Developmental Activities
    • 9.3.3. Clinical Development
    • 9.3.4. Safety and Efficacy
    • 9.3.5. Product Profile
  • 9.4. Trappsol Cyclo (Cyclodextrin): CTD Holdings
    • 9.4.1. Drug Description
    • 9.4.2. Other Developmental Activities
    • 9.4.3. Clinical Development
    • 9.4.4. Safety and Efficacy
    • 9.4.5. Product Profile
  • 9.5. IB1001: IntraBio
    • 9.5.1. Drug Description
    • 9.5.2. Other Developmental Activities
    • 9.5.3. Clinical Development
    • 9.5.4. Product Profile

10. Niemann Pick Disease Type C: 7 Major Market Analysis

  • 10.1. Key Findings
  • 10.2. Total Market Size of Niemann Pick Disease Type C in 7MM
  • 10.3. Market Size by Therapeutic Class in 7MM
  • 10.4. Market Size by Therapies in 7MM

11. The United States: Market Outlook

  • 11.1. United States Market Size
    • 11.1.1. Total Market size of Niemann Pick Disease Type C [NPC]
    • 11.1.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 11.1.3. Market Size by Therapies

12. EU-5 Countries: Market Outlook

  • 12.1. Germany
    • 12.1.1. Total Market size of Niemann Pick Disease Type C
    • 12.1.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 12.1.3. Market Size by Therapies
  • 12.2. France
    • 12.2.1. Total Market size of Niemann Pick Disease Type C
    • 12.2.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 12.2.3. Market Size by Therapies
  • 12.3. Italy
    • 12.3.1. Total Market size of Niemann Pick Disease Type C
    • 12.3.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 12.3.3. Market Size by Therapies
  • 12.4. Spain
    • 12.4.1. Total Market size of Niemann Pick Disease Type C
    • 12.4.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 12.4.3. Market Size by Therapies
  • 12.5. United Kingdom
    • 12.5.1. Total Market size of Niemann Pick Disease Type C
    • 12.5.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 12.5.3. Market Size by Therapies

13. Japan: Market Outlook

    • 13.1.1. Total Market size of Niemann Pick Disease Type C
    • 13.1.2. Niemann Pick Disease Type C Market by Therapeutic Class
    • 13.1.3. Market Size by Therapies

14. Market Drivers

15. Market Barriers

16. Appendix

  • 16.1. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

19. About DelveInsight

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