Niemann Pick Type C Disease - Pipeline Analysis 2019
- UNI4065857
- 79 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 present for NPC in the U.S. 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. 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.
Scope of the report:
• 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 (NPC) pipeline 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 pipeline 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 8
1.2 Secondary Research 8
1.3 Scope of the report: 8
2. DISEASE OVERVIEW 9
2.1 Introduction 9
2.2 Classification of Niemann-Pick Disease 10
2.2.1 Niemann-Pick disease type A 10
2.2.2 Niemann-Pick disease type B 10
2.2.3 Niemann-Pick disease type C 10
2.3 Classification of NPC 10
2.4 Signs and Symptoms 12
2.5 Causes 14
2.5.1 NPC1 and NPC2 proteins 14
2.6 Pathogenesis 15
2.6.1 Molecular Pathogenesis 15
2.6.2 NPC1 15
2.6.3 NPC2 16
2.7 Pathophysiology 17
2.7.1 Cell Death and Oxidative Damage in NPC 18
2.7.2 Increase in Mitochondrial Cholesterol and Dysfunction in NPC Disease 20
2.7.3 Peroxisomes Alterations in NPC Disease 20
2.7.4 Intracellular Accumulation of Vitamin E in NPC Disease 21
2.7.5 Glycosphingolipid Accumulation in NPC Disease 22
2.7.6 Other Sources of Reactive Species in Oxidative Damage in NPC 23
2.8 Diagnosis 24
2.8.1 Biochemical Testing: 25
2.8.2 Histology: 25
2.8.3 Molecular Genetic Testing: 25
2.8.4 Testing Strategy 27
2.8.5 Biomarkers: 27
2.8.5.1 Oxysterols (cholesterol oxidation products) 27
2.8.5.2 Lyso-sphingolipids 28
2.8.5.3 Bile acids 28
2.8.6 The Filipin test 28
2.8.7 Brain imaging 29
2.8.8 Differential Diagnosis 29
2.8.8.1 Neonatal and infantile presentations 29
2.8.8.2 Childhood presentations 29
2.8.8.3 Adolescent and adult presentations 30
2.9 Diagnosis Algorithm 33
2.10 Treatment 34
2.10.1 Symptomatic therapy 35
2.10.2 Evaluations Following Initial Diagnosis 37
2.10.3 Treatment of Manifestations 37
2.10.4 Prevention of Secondary Complications 37
2.10.5 Surveillance 38
2.10.6 Agents/Circumstances to Avoid 38
2.10.7 Evaluation of Relatives at Risk 38
2.10.8 Therapies Under Investigation 38
2.10.9 Other 39
2.11 Genetic Counselling 39
2.12 Treatment Algorithm 40
3. PIPELINE THERAPEUTICS AT A GLANCE 41
3.1 Products Under Development by Technology 41
4. COMPARATIVE ANALYSIS 42
4.1 Number of Products in NPC 42
5. LATE PHASE PRODUCTS (PHASE III) 43
5.1 Comparative Analysis 43
5.2 Arimoclomol: Orphazyme 43
5.2.1 Product Description 43
5.2.2 Research and Development 44
5.2.2.1 Clinical Studies 44
5.2.2.1.1 Phase II/III 44
5.2.2.1.2 Results of Analysis 44
5.2.2.2 Observational Study 46
5.2.2.3 Results of Analysis 46
5.2.3 Product Development Activities 46
5.2.3.1 Agreement 46
5.2.3.2 Designation 47
5.3 Trappsol Cyclo: CTD Holdings 48
5.3.1 Product Description 48
5.3.2 Research and Development 48
5.3.2.1 Clinical Studies 48
5.3.2.1.1 Phase II/III 48
5.3.2.1.2 Results of Analysis 48
5.3.3 Product Development Activities 50
5.3.3.1 Designation 50
5.3.3.2 Clinical Trial Application Approval 51
5.3.3.3 Compassionate Use 51
5.4 Adrabetadex: Mallinckrodt Pharmaceuticals 52
5.4.1 Product Description 52
5.4.2 Research and Development 53
5.4.2.1 Clinical Studies 53
5.4.2.1.1 Phase IIb/III 53
5.4.2.1.2 Results of Analysis 54
5.4.3 Product Development Activities 54
5.4.3.1 FDA Type A Meeting 54
5.4.3.2 Agreement 54
5.4.3.3 Funding 55
5.4.3.4 Designation 55
5.4.3.5 Patent 56
6. MID PHASE PRODUCTS (PHASE II) 57
6.1 Comparative Analysis 57
6.2 IB1001: IntraBio 57
6.2.1 Product Description 57
6.2.2 Research and Development 57
6.2.2.1 Clinical Studies 57
6.2.2.1.1 Phase II 58
6.2.3 Product Development Activities 58
6.2.3.1 Designation 58
7. PRE-CLINICAL AND DISCOVERY STAGE PRODUCTS 60
7.1 Comparative Analysis 60
7.2 Ursodeoxycholic acid: IntraBio 60
7.2.1 Product Description 60
7.2.2 Research and Development 61
7.2.2.1 Observational Study 61
7.2.3 Product Development Activities 61
7.2.3.1 Designation 61
7.3 PERL101: Perlara 62
7.3.1 Product Description 62
7.3.2 Research and Development 62
7.3.2.1 Preclinical Studies 62
7.3.3 Product Development Activities 62
7.3.3.1 Technology 62
7.3.3.2 Collaboration 62
7.4 Bryostatin-1: Neurotrope BioScience 63
7.4.1 Product Description 63
7.4.1.1 Agreement 64
7.5 Exosomes: Evox Therapeutics 65
7.5.1 Product Description 65
7.5.2 Product Development Activities 65
7.5.2.1 Funding 65
7.5.2.2 Financing 65
7.5.2.3 Technology 65
7.5.2.4 Patent 66
7.6 Gene therapy for NPC: Amicus Therapeutics 67
7.6.1 Product Description 67
7.6.2 Product Development Activities 67
7.6.2.1 Agreement 67
7.7 Cyclodextrins: Okklo Life Sciences 68
7.7.1 Product Description 68
7.7.2 Product Development Activities 68
7.7.2.1 Technology 68
7.7.2.2 Agreement 68
8. THERAPEUTIC ASSESSMENT 70
8.1 Assessment by Stage and Product Type 70
8.2 Assessment by Route of Administration 70
8.3 Assessment by Stage and Route of Administration 71
8.4 Assessment by Molecule Type 72
8.5 Assessment by Stage and Molecule Type 72
9. APPROVAL TIMELINES 74
9.1 Approval Timelines for Clinical Products 74
10. ANALYST INSIGHTS 75
Table 2: NPC1 Variants 16
Table 3: NPC2 Pathogenic Variants 17
Table 4: Molecular Genetic Testing Used in Niemann-Pick Disease Type C 27
Table 5: Clinical Severity assessment 31
Table 6: Multidisciplinary assessments of patients with NPC 34
Table 7: Recommended assessments 35
Table 8: Total Number of Products in NPC 42
Table 9: Late Stage Products (Phase III) 43
Table 10: Full Data Set Results of the trial 45
Table 11: Clinical Trial Description: Arimoclomol 46
Table 12: General Description: Arimoclomol 47
Table 13: Clinical Trial Description: Trappsol Cyclo 50
Table 14: General Description: Trappsol Cyclo 52
Table 15: Clinical Trial Description: VTS-270 54
Table 16: Patent Details: VTS-270 56
Table 17: General Description: Adrabetadex 56
Table 18: Mid Stage Products (Phase II) 57
Table 19: Clinical Trial Description: IB1001 58
Table 20: General Description: IB1001 58
Table 21: Pre-clinical and Discovery Stage Products 60
Table 22: General Description: Ursodeoxycholic Acid 61
Table 23: General Description: PERL101 63
Table 24: General Description: Bryostatin-1 64
Table 25: General Description: Exosomes 66
Table 26: General Description: Gene therapy for NPC 67
Table 27: General Description: Cyclodextrins 69
Table 28: Assessment by Stage and Product Type 70
Table 29: Assessment by Route of Administration 71
Table 30: Assessment by Stage and Route of Administration 71
Table 31: Assessment by Molecule Type 72
Table 32: Assessment by Stage and Molecule Type 73
Table 33: Approval Timelines for Clinical Products 74
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