Niemann-Pick Disease Type C (NPC) Therapeutics - Pipeline Analysis 2019, Clinical Trials and Results, Patents, Designations, Collaborations and Other Developments

Chapter 1. Research Background

1.1 Research Objectives

1.2 Definition

1.3 Research Scope

1.3.1 Pipeline Drugs by Phase

1.3.2 Pipeline Drugs by Route of Administration

1.3.3 Pipeline Drugs by Molecule Type

1.4 Key Stakeholders

Chapter 2. Research Methodology

2.1 Secondary Research

2.2 Primary Research

2.2.1 Breakdown of Primary Research Respondents

2.2.1.1 By industry participant

2.2.1.2 By geography

2.2.1.3 By designation

2.2.2 Key Opinion Leaders (KOLs) Validation

Chapter 3. Executive Summary

3.1 Voice of KOLs

Chapter 4. Introduction

4.1 Disease Overview

4.2 Causes and Risk Factors

4.3 Sign and Symptoms

4.4 Diagnosis

4.5 Treatment

4.6 Key Drivers

4.6.1 Increasing Incidence of NPC

4.6.2 Lack of Effective Therapies

4.6.3 Rising R&D Investments

4.7 Key Barriers

4.7.1 High Cost of Therapy

4.7.2 High Chances of Toxicities and Side-effects

4.8 Technology Overview

4.9 Drug Designations

Chapter 5. NPC Epidemiology Forecast for 7MM

5.1 Epidemiology Forecast for 7MM

5.1.1 By Type

5.2 Epidemiology Forecast for the U.S.

5.2.1 By Type

5.3 Epidemiology Forecast for EU5

5.3.1 By Type

5.3.2 By Country

5.3.2.1 Germany

5.3.2.1.1 By type

5.3.2.2 France

5.3.2.2.1 By type

5.3.2.3 U.K.

5.3.2.3.1 By type

5.3.2.4 Italy

5.3.2.4.1 By type

5.3.2.5 Spain

5.3.2.5.1 By type

5.4 Epidemiology Forecast for Japan

5.4.1 By Type

Chapter 6. Pipeline Drugs Outlook

6.1 NPC Pipeline Drugs Snapshot

6.1.1 Pipeline Drugs Snapshot by Phase

6.1.2 Pipeline Drugs Snapshot by Route of Administration

6.1.3 Pipeline Drugs Snapshot by Molecule Type

6.1.4 Pipeline Drugs Snapshot by Company

Chapter 7. NPC Therapeutics Marketed Drug Analysis

7.1 Marketed Drug Profile

7.1.1 Zavesca

7.1.1.1 Product description

7.1.1.2 Dosage

7.1.1.3 Regulatory milestones

7.1.1.4 Clinical trials and results

7.1.1.5 Patent

7.2 Challenges Faced by Marketed Drug Manufacturer

7.2.1 Low Incidence of the Disease

7.2.2 High Cost of Drugs

Chapter 8. NPC Therapeutics Pipeline Drugs Analysis

8.1 Phase II/III Drugs

8.1.1 Arimoclomol

8.1.1.1 Clinical trials

8.1.1.2 Clinical trial results

8.1.1.3 Technology

8.1.1.4 Financing

8.1.1.5 NDA filing

8.1.1.6 Designations

8.1.1.7 Strategic developments

8.1.1.8 Patents

8.1.2 Xxxx

8.1.2.1 Clinical trials

8.1.2.2 Clinical trial results

8.1.2.3 Patents

8.1.2.4 Spin-off

8.1.2.5 Financing

8.1.2.6 Designations

8.1.2.7 Strategic developments

8.2 Phase II Drugs

8.2.1 IB1001

8.2.1.1 Clinical trials

8.2.1.2 IND application

8.2.1.3 Designations

8.3 Phase I/II Drugs

8.3.1 Trappsol Cyclo

8.3.1.1 Clinical trials

8.3.1.2 Clinical trial results

8.3.1.3 Designations

8.3.1.4 Financing

8.4 Pre-clinical

8.4.1 Bryostatin-1

8.4.1.1 Pre-clinical results

8.4.1.2 Strategic developments

8.4.2 Xxxx

8.4.2.1 Spin off

8.4.2.2 Strategic developments

8.4.3 Xxxx

8.5 Discovery

8.5.1 Cyclodextrins

8.5.2 Xxxx

8.5.2.1 Strategic developments

8.5.3 Xxxx

8.6 Inactive

8.6.1 Xxxx

8.6.1.1 Designations

Chapter 9. Clinical Trials Review

9.1 Clinical Trials by Geography

9.2 Clinical Trials by Trial Status

Chapter 10. Regulatory Framework for Drug Approval

10.1 U.S.

10.2 Europe

10.2.1 Centralized Procedure

10.2.2 Mutual Recognition Procedure

10.2.3 Decentralized Procedure

10.2.4 Nationalized Procedures

10.3 Japan

Chapter 11. Competitive Landscape

11.1 Summary of Strategic Development Activities

11.2 Attribute Analysis

11.3 Comparative Analysis

11.4 Expected Launch Time of Phase II/III Clinical Stage Products

11.5 Key Winning Strategies

11.5.1 Development through Subsidiaries

11.5.2 Acquisition of Additional Pipeline Products

11.6 Pharma Proff’s View

Chapter 12. Company Profiles

12.1 Orphazyme A/S

12.1.1 Business Overview

12.1.2 Product and Service Offerings

12.1.2.1 Pipeline products

12.2 Xxxx

12.2.1 Business Overview

12.2.2 Product and Service Offerings

12.2.2.1 Pipeline products

12.3 Xxxx

12.3.1 Business Overview

12.3.2 Product and Service Offerings

12.3.2.1 Pipeline products

12.4 Xxxx

12.4.1 Business Overview

12.4.2 Product and Service Offerings

12.4.2.1 Marketed products

12.4.2.2 Pipeline products

12.5 Xxxx

12.5.1 Business Overview

12.5.2 Product and Service Offerings

12.5.2.1 Pipeline products

12.6 Xxxx

12.6.1 Business Overview

12.6.2 Product and Service Offerings

12.6.2.1 Pipeline products

12.7 Xxxx

12.7.1 Business Overview

12.7.2 Product and Service Offerings

12.7.2.1 Marketed products

12.7.2.2 Pipeline products

12.8 Xxxx

12.8.1 Business Overview

12.8.2 Product and Service Offerings

12.8.2.1 Pipeline products

12.9 Xxxx

12.9.1 Business Overview

12.9.2 Product and Service Offerings

12.9.2.1 Marketed product

12.9.2.2 Pipeline products

Chapter 13. Appendix

13.1 Recently Organized/Held Seminars and Conferences

13.2 Upcoming Conferences

13.3 Sources and References

13.4 Abbreviations

13.5 Related Reports

 

LIST OF TABLES

 

TABLE 1 NPC EPIDEMIOLOGY FOR 7MM (2016–2021)

TABLE 2 NPC EPIDEMIOLOGY FOR 7MM (2022–2028)

TABLE 3 DRUG DESIGNATIONS

TABLE 4 NPC EPIDEMIOLOGY IN 7MM (2016–2021)

TABLE 5 NPC EPIDEMIOLOGY IN 7MM (2022–2028)

TABLE 6 NPC INCIDENT CASES, BY TYPE FOR 7MM (2016–2021)

TABLE 7 NPC INCIDENT CASES, BY TYPE FOR 7MM (2022–2028)

TABLE 8 NPC EPIDEMIOLOGY IN THE U.S. (2016–2021)

TABLE 9 NPC EPIDEMIOLOGY IN THE U.S. (2022–2028)

TABLE 10 NPC INCIDENT CASES, BY TYPE FOR THE U.S. (2016–2021)

TABLE 11 NPC INCIDENT CASES, BY TYPE FOR THE U.S. (2022–2028)

TABLE 12 NPC EPIDEMIOLOGY IN EU5 (2016–2021)

TABLE 13 NPC EPIDEMIOLOGY IN EU5 (2022–2028)

TABLE 14 NPC INCIDENT CASES, BY TYPE FOR EU5 (2016–2021)

TABLE 15 NPC INCIDENT CASES, BY TYPE FOR EU5 (2022–2028)

TABLE 16 NPC EPIDEMIOLOGY IN GERMANY (2016–2021)

TABLE 17 NPC EPIDEMIOLOGY IN GERMANY (2022–2028)

TABLE 18 NPC INCIDENT CASES, BY TYPE FOR GERMANY (2016–2021)

TABLE 19 NPC INCIDENT CASES, BY TYPE FOR GERMANY (2022–2028)

TABLE 20 NPC EPIDEMIOLOGY IN FRANCE (2016–2021)

TABLE 21 NPC EPIDEMIOLOGY IN FRANCE (2022–2028)

TABLE 22 NPC INCIDENT CASES BY TYPE FOR FRANCE (2016–2021)

TABLE 23 NPC INCIDENT CASES BY TYPE FOR FRANCE (2022–2028)

TABLE 24 NPC EPIDEMIOLOGY IN THE U.K. (2016–2021)

TABLE 25 NPC EPIDEMIOLOGY IN THE U.K. (2022–2028)

TABLE 26 NPC INCIDENT CASES, BY TYPE FOR THE U.K. (2016–2021)

TABLE 27 NPC INCIDENT CASES, BY TYPE FOR THE U.K (2022–2028)

TABLE 28 NPC EPIDEMIOLOGY IN ITALY (2016–2021)

TABLE 29 NPC EPIDEMIOLOGY IN ITALY (2022–2028)

TABLE 30 NPC INCIDENT CASES, BY TYPE FOR ITALY (2016–2021)

TABLE 31 NPC INCIDENT CASES, BY TYPE FOR ITALY (2022–2028)

TABLE 32 NPC EPIDEMIOLOGY IN SPAIN (2016–2021)

TABLE 33 NPC EPIDEMIOLOGY IN SPAIN (2022–2028)

TABLE 34 NPC INCIDENT CASES, BY TYPE FOR SPAIN (2016–2021)

TABLE 35 NPC INCIDENT CASES, BY TYPE FOR SPAIN (2022–2028)

TABLE 36 NPC EPIDEMIOLOGY IN JAPAN (2016–2021)

TABLE 37 NPC EPIDEMIOLOGY IN JAPAN (2022–2028)

TABLE 38 NPC INCIDENT CASES, BY TYPE FOR JAPAN (2016–2021)

TABLE 39 NPC INCIDENT CASES, BY TYPE FOR JAPAN (2022–2028)

TABLE 40 DESCRIPTION OF ZAVESCA

TABLE 41 DOSAGE REGIMENS FOR PATIENTS BELOW 12 YEARS OF AGE

TABLE 42 PATENT FOR ZAVESCA:

TABLE 43 DESCRIPTION OF ARIMOCLOMOL

TABLE 44 CLINICAL TRIALS OF ARIMOCLOMOL

TABLE 45 ARIMOCLOMOL PATENTS

TABLE 46 DESCRIPTION OF XXXX

TABLE 47 CLINICAL TRIALS OF XXXX

TABLE 48 DESCRIPTION OF IB1001

TABLE 49 CLINICAL TRIALS OF IB1001

TABLE 50 DESCRIPTION OF TRAPPSOL CYCLO

TABLE 51 CLINICAL TRIALS OF TRAPPSOL CYCLO

TABLE 52 DESCRIPTION OF BRYOSTATIN-1

TABLE 53 DESCRIPTION OF CYCLODEXTRINS

TABLE 54 DESCRIPTION OF XXXX

TABLE 55 COLLABORATIONS AND LICENSING AGREEMENTS

TABLE 56 ACQUISITIONS

TABLE 57 ATTRIBUTE ANALYSIS – XXXX AND XXXX

TABLE 58 COMPARISON OF MARKETED AND PHASE II/III DRUGS FOR NPC, BASED ON EFFICACY

TABLE 59 EXPECTED LAUNCH TIME OF PHASE II/III CLINICAL STAGE PRODUCTS

TABLE 60 ORPHAZYME A/S – AT A GLANCE

TABLE 61 PIPELINE PRODUCTS

TABLE 62 INTRABIO – AT A GLANCE

TABLE 63 XXXX – AT A GLANCE

TABLE 64 XXXX – AT A GLANCE

TABLE 65 MARKETED PRODUCTS

TABLE 66 PIPELINE PRODUCTS

TABLE 67 XXXX – AT A GLANCE

TABLE 68 PIPELINE PRODUCTS

TABLE 69 NEUROTROPE INC. – AT A GLANCE

TABLE 70 PIPELINE PRODUCTS

TABLE 71 ACTELION PHARMACEUTICALS LTD – AT A GLANCE

TABLE 72 MARKETED PRODUCTS

TABLE 73 PIPELINE PRODUCTS

TABLE 74 XXXX– AT A GLANCE

TABLE 75 AMICUS THERAPEUTICS INC. – AT A GLANCE

TABLE 76 PIPELINE PRODUCTS

TABLE 77 RECENTLY ORGANIZED SEMINARS AND CONFERENCES

TABLE 78 UPCOMING SEMINARS AND CONFERENCES

TABLE 79 SOURCES & REFERENCES

TABLE 80 ABBREVIATIONS

 

LIST OF FIGURES

 

FIG 1 RESEARCH METHODOLOGY

FIG 2 BREAKDOWN OF PRIMARY RESEARCH RESPONDENTS, BY INDUSTRY PARTICIPANT

FIG 3 BREAKDOWN OF PRIMARY RESEARCH, BY GEOGRAPHY

FIG 4 BREAKDOWN OF PRIMARY RESEARCH, BY DESIGNATION

FIG 5 NPC INCIDENT CASES, BY TYPE FOR 7MM (2016–2028)

FIG 6 TOTAL NUMBER OF INCIDENT CASES OF NPC IN 7MM (2016–2028)

FIG 7 NPC INCIDENT CASES, BY TYPE FOR 7MM (2016–2028)

FIG 8 NPC EPIDEMIOLOGY IN THE U.S. (2016–2028)

FIG 9 NPC INCIDENT CASES, BY TYPE FOR THE U.S. (2016–2028)

FIG 10 NPC EPIDEMIOLOGY IN EU5 (2016–2028)

FIG 11 NPC INCIDENT CASES, BY TYPE FOR EU5 (2016–2028)

FIG 12 NPC EPIDEMIOLOGY IN GERMANY (2016–2028)

FIG 13 NPC INCIDENT CASES, BY TYPE FOR GERMANY (2016–2028)

FIG 14 NPC EPIDEMIOLOGY IN FRANCE (2016–2028)

FIG 15 NPC CASES BY TYPE FOR FRANCE (2016–2028)

FIG 16 NPC EPIDEMIOLOGY IN THE U.K. (2016–2028)

FIG 17 NPC INCIDENT CASES, BY TYPE FOR THE U.K. (2016–2028)

FIG 18 NPC EPIDEMIOLOGY IN ITALY (2016–2028)

FIG 19 NPC INCIDENT CASES, BY TYPE FOR ITALY (2016–2028)

FIG 20 NPC EPIDEMIOLOGY IN SPAIN (2016–2028)

FIG 21 NPC INCIDENT CASES, BY TYPE FOR SPAIN (2016–2028)

FIG 22 NPC EPIDEMIOLOGY IN JAPAN (2016–2028)

FIG 23 NPC INCIDENT CASES, BY TYPE FOR JAPAN (2016–2028)

FIG 24 NPC PIPELINE DRUGS SNAPSHOT, BY PHASE (2019)

FIG 25 NPC PIPELINE DRUGS SNAPSHOT, BY ROUTE OF ADMINISTRATION (2019)

FIG 26 NPC THERAPEUTICS PIPELINE DRUGS SNAPSHOT, BY MOLECULE TYPE (2019)

FIG 27 COMPANY BENCHMARKING BASED ON DRUG CANDIDATES

FIG 28 BREAKDOWN OF CLINICAL TRIALS, BY GEOGRAPHY

FIG 29 BREAKDOWN OF CLINICAL TRIALS, BY TRIAL STATUS

FIG 30 DRUG DISCOVERY AND APPROVAL PROCESS (PRE-CLINICAL AND CLINICAL TRIALS STAGES)

FIG 31 DRUG APPROVAL PROCESS (NDA REVIEW AND POST-MARKETING STAGES)

FIG 32 NEW DRUG DEVELOPMENT AND APPROVAL PROCESS IN EU

FIG 33 MARKETING AUTHORIZATION PROCEDURES IN EU

FIG 34 CENTRALIZED PROCEDURE FOR DRUG APPROVAL

FIG 35 MUTUAL RECOGNITION PROCEDURE FOR DRUG APPROVAL

FIG 36 DECENTRALIZED PROCEDURE FOR DRUG APPROVAL

FIG 37 NEW DRUG DEVELOPMENT AND APPROVAL PROCESS in japan

FIG 38 EXPECTED LAUNCH TIME