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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.
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 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. epidemiology model for NPC estimates that NPC1 subtype is the most prevalent in the 7MM countries, as compared to NPC2 subtype. 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 analysts, the total diagnosed prevalent population of NPC in the 7MM was assessed to be 1,058 in 2017.
According to 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 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.
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.
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 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. epidemiology model for NPC estimates that NPC1 subtype is the most prevalent in the 7MM countries, as compared to NPC2 subtype. 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 analysts, the total diagnosed prevalent population of NPC in the 7MM was assessed to be 1,058 in 2017.
According to 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 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.
Frequently Asked Questions
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The report provides a detailed evaluation of the market by highlighting information on different aspects which include drivers, restraints, opportunities, and threats. This information can help stakeholders to make appropriate decisions before investing.
