Niemann Pick Type C Disease - Competitive Analysis 2019
- UNI4065858
- 86 Pages
- June 2019
- Healthcare
Niemann Pick Type-C Disease (NPC) is a progressive autosomal recessive genetic disorder of lysosome storage due to an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. The accumulation of these substances damages the affected areas. It 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. It affects both males and females equally. NPC is estimated to occur in 1 in 100,000-120,000 live births.
The onset of symptoms may begin at any age, most commonly in school age children; however, infants, adolescents and adults can all be affected by the disease. With the progression of disease, children may experience developmental delays, challenges with motor skills (e.g. increased clumsiness and abnormal posturing) and difficulty in making vertical eye movements. Dystonia and seizures are common in the patients suffering from NPC.
Several tests are available for the diagnosis of NPC. These tests include biochemical testing, molecular genetic testing, NPC screening using biomarkers among others. No marketed drugs are currently available for NPC in the U.S and only one marketed drug i.e. Miglustat is available in European Union (EU) and Japan. The drug development companies are involved in quest to develop an effective drug and better treatment strategies. There are several companies utilizing their proprietary technologies for the development of effective and better drugs.
The therapeutic pipeline of NPC Disease consists of approximately 9+ products in different stages of development along with single marketed drug. Currently 2+ drugs are in Phase III development and major drugs are in pre-clinical and discovery stage.
Top Company Analysed
Some of the key players include Orphazyme, CTD Holdings, Mallinckrodt Pharmaceuticals, IntraBio, Perlara, Neurotrope BioScience, Evox Therapeutics, Okklo Life Sciences, Amicus Therapeutics and Johnson & Johnson.
Scope of the report:
• The report provides the marketed drugs information including its approval details, development activities and details of patent expiry
• Provides an overview of therapeutic pipeline activity for Niemann Pick Type-C Disease across the complete product development cycle including all clinical and non-clinical stages
• The report comprises of detailed description of Niemann Pick Type-C Disease therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
• Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
Reasons to buy:
• The Niemann Pick Type-C Disease competitive report presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
• The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
• The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
• Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
• Examination of industry attractiveness with the help of approval timelines
• The study comprehensively covers the market across drugs in different phases of development
Customization Options:
The Niemann Pick Type-C Disease competitive analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.
1.1 Objective of the Study 9
1.2 Secondary Research 9
1.3 Scope of the report: 9
2. DISEASE OVERVIEW 10
2.1 Introduction 10
2.2 Classification of Niemann-Pick Disease 11
2.2.1 Niemann-Pick disease type A 11
2.2.2 Niemann-Pick disease type B 11
2.2.3 Niemann-Pick disease type C 11
2.3 Classification of NPC 11
2.4 Signs and Symptoms 13
2.5 Causes 15
2.5.1 NPC1 and NPC2 proteins 15
2.6 Pathogenesis 16
2.6.1 Molecular Pathogenesis 16
2.6.2 NPC1 16
2.6.3 NPC2 17
2.7 Pathophysiology 18
2.7.1 Cell Death and Oxidative Damage in NPC 19
2.7.2 Increase in Mitochondrial Cholesterol and Dysfunction in NPC Disease 21
2.7.3 Peroxisomes Alterations in NPC Disease 21
2.7.4 Intracellular Accumulation of Vitamin E in NPC Disease 22
2.7.5 Glycosphingolipid Accumulation in NPC Disease 23
2.7.6 Other Sources of Reactive Species in Oxidative Damage in NPC 24
2.8 Diagnosis 25
2.8.1 Biochemical Testing: 26
2.8.2 Molecular Genetic Testing: 26
2.8.3 Testing Strategy 28
2.8.4 Biomarkers 28
2.8.4.1 Oxysterols (cholesterol oxidation products) 29
2.8.4.2 Lyso-sphingolipids 29
2.8.4.3 Bile acids 29
2.8.5 The Filipin test 29
2.8.6 Brain imaging 30
2.8.7 Differential Diagnosis 30
2.8.7.1 Neonatal and infantile presentations 30
2.8.7.2 Childhood presentations 30
2.8.7.3 Adolescent and adult presentations 31
2.9 Diagnosis Algorithm 34
2.10 Treatment 35
2.10.1 Symptomatic therapy 36
2.10.2 Evaluations Following Initial Diagnosis 38
2.10.3 Treatment of Manifestations 38
2.10.4 Prevention of Secondary Complications 38
2.10.5 Surveillance 39
2.10.6 Agents/Circumstances to Avoid 39
2.10.7 Evaluation of Relatives at Risk 39
2.10.8 Therapies Under Investigation 39
2.10.9 Other 40
2.11 Genetic Counselling 40
2.12 Treatment Algorithm 41
3. PRODUCTS AND COMPANY CONSIDERED 42
3.1 Products Under Development by Technology 42
3.2 Company Considered in the Study 42
4. PRODUCTS UNDER DEVELOPMENT BY DESIGNATION 43
4.1 Products Under Development by Designation 43
5. MARKETED AND EMERGING PRODUCTS 44
5.1 Number of Marketed and Emerging Products 44
5.2 Marketed Product 44
5.2.1 Zavesca: Johnson & Johnson 44
5.2.1.1 Product Description 44
5.2.1 Regulatory Milestones 46
5.2.1.1 Approval 46
5.2.2 Research and Development 46
5.2.2.1 Clinical Studies 46
5.2.2.1.1 Phase II 46
5.2.2.1.2 Results of Analysis 46
5.2.3 Product Development Activities 48
5.2.3.1 Acquisition 48
5.2.3.2 Designation 48
6. PIPELINE THERAPEUTICS AT A GLANCE 49
6.1 Pipeline Therapeutics at a Glance 49
7. COMPARATIVE ANALYSIS 50
7.1 Number of Products in NPC 50
8. LATE PHASE PRODUCTS (PHASE III) 51
8.1 Comparative Analysis 51
8.2 Arimoclomol: Orphazyme 51
8.2.1 Product Description 51
8.2.2 Research and Development 52
8.2.2.1 Clinical Studies 52
8.2.2.1.1 Phase II/III 52
8.2.2.1.2 Results of Analysis 52
8.2.2.2 Observational Study 54
8.2.2.2.1 Results of Analysis 54
8.2.3 Product Development Activities 54
8.2.3.1 Agreement 54
8.2.3.2 Designation 55
8.3 Trappsol Cyclo: CTD Holdings 56
8.3.1 Product Description 56
8.3.2 Research and Development 56
8.3.2.1 Clinical Studies 56
8.3.2.1.1 Phase II/III 56
8.3.2.1.2 Results of Analysis 56
8.3.3 Product Development Activities 58
8.3.3.1 Designation 58
8.3.3.2 Clinical Trial Application Approval 59
8.3.3.3 Compassionate Use 59
8.4 Adrabetadex: Mallinckrodt Pharmaceuticals 60
8.4.1 Product Description 60
8.4.2 Research and Development 61
8.4.2.1 Clinical Studies 61
8.4.2.1.1 Phase IIb/III 61
8.4.2.1.2 Results of Analysis 62
8.4.3 Product Development Activities 62
8.4.3.1 FDA Type A Meeting 62
8.4.3.2 Agreement 62
8.4.3.3 Funding 63
8.4.3.4 Designation 63
8.4.3.5 Patent 64
9. MID PHASE PRODUCTS (PHASE II) 65
9.1 Comparative Analysis 65
9.2 IB1001: IntraBio 65
9.2.1 Product Description 65
9.2.2 Research and Development 65
9.2.2.1 Clinical Studies 65
9.2.2.1.1 Phase II 66
9.2.3 Product Development Activities 66
9.2.3.1 Designation 66
10. PRE-CLINICAL AND DISCOVERY STAGE PRODUCTS 68
10.1 Comparative Analysis 68
10.2 Ursodeoxycholic acid: IntraBio 68
10.2.1 Product Description 68
10.2.2 Research and Development 69
10.2.2.1 Observational Study 69
10.2.3 Product Development Activities 69
10.2.3.1 Designation 69
10.3 PERL101: Perlara 70
10.3.1 Product Description 70
10.3.2 Research and Development 70
10.3.2.1 Preclinical Studies 70
10.3.3 Product Development Activities 70
10.3.3.1 Technology 70
10.3.3.2 Collaboration 70
10.4 Bryostatin-1: Neurotrope BioScience 71
10.4.1 Product Description 71
10.4.1.1 Agreement 72
10.5 Exosomes: Evox Therapeutics 73
10.5.1 Product Description 73
10.5.2 Product Development Activities 73
10.5.2.1 Funding 73
10.5.2.2 Financing 73
10.5.2.3 Technology 73
10.5.2.4 Patent 74
10.6 Gene therapy for NPC: Amicus Therapeutics 75
10.6.1 Product Description 75
10.6.2 Product Development Activities 75
10.6.2.1 Agreement 75
10.7 Cyclodextrins: Okklo Life Sciences 76
10.7.1 Product Description 76
10.7.2 Product Development Activities 76
10.7.2.1 Technology 76
10.7.2.2 Agreement 76
11. THERAPEUTIC ASSESSMENT 78
11.1 Assessment by Stage and Product Type 78
11.2 Assessment by Route of Administration 78
11.3 Assessment by Stage and Route of Administration 79
11.4 Assessment by Molecule Type 80
11.5 Assessment by Stage and Molecule Type 80
12. APPROVAL TIMELINES 82
12.1 Approval Timelines for Clinical Products 82
13. ANALYST INSIGHTS 83
Table 2: NPC1 Variants 17
Table 3: NPC2 Pathogenic Variants 18
Table 4: Molecular Genetic Testing Used in Niemann-Pick Disease Type C 28
Table 5: Clinical Severity Assessment 32
Table 6: Multidisciplinary assessments of patients with NPC 35
Table 7: Recommended assessments 36
Table 8: Total Number of Marketed and Emerging Products 44
Table 9: Dosing in Patients Under 12 Years 45
Table 10: Clinical Trial Description: Miglustat 48
Table 11: General Description: Miglustat 48
Table 12: Total Number of Products in NPC 50
Table 13: Late Stage Products (Phase III) 51
Table 14: Full Data Set Results of the trial 53
Table 15: Clinical Trial Description: Arimoclomol 54
Table 16: General Description: Arimoclomol 55
Table 17: Clinical Trial Description: Trappsol Cyclo 58
Table 18: General Description: Trappsol Cyclo 60
Table 19: Clinical Trial Description: VTS-270 62
Table 20: Patent Details: VTS-270 64
Table 21: General Description: Adrabetadex 64
Table 22: Mid Stage Products (Phase II) 65
Table 23: Clinical Trial Description: IB1001 66
Table 24: General Description: IB1001 66
Table 25: Pre-clinical and Discovery Stage Products 68
Table 26: General Description: Ursodeoxycholic Acid 69
Table 27: General Description: PERL101 71
Table 28: General Description: Bryostatin-1 72
Table 29: General Description: Exosomes 75
Table 30: General Description: Gene therapy for NPC 76
Table 31: General Description: Cyclodextrins 78
Table 32: Assessment by Stage and Product Type 79
Table 33: Assessment by Route of Administration 80
Table 34: Assessment by Stage and Route of Administration 80
Table 35: Assessment by Molecule Type 81
Table 36: Assessment by Stage and Molecule Type 82
Table 37: Approval Timelines for Clinical Products 83
1.
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