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Global Friedreich Ataxia Drug Pipeline Trends 2019: Discovery, Pre-clinical, Clinical, In Approval Therapeutics, Companies and Markets

August 2019 | 82 pages | ID: G38E952CFBFEN
VPAResearch

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High levels of pipeline activity are being observed in Friedreich Ataxia treatment during 2019. Clinical development activities are being undertaken by more than 20 companies including Adverum Biotechnologies Inc, Agios Pharmaceuticals Inc, Amylyx Pharmaceutical Corp, BioElectron Technology Corp , BioMarin Pharmaceutical Inc and others.

A Significant contribution to the Friedreich Ataxia pipeline is being observed from emerging companies opting for new approaches to clinical development strategy. The early stage and late-stage Friedreich Ataxia pipeline included 20 compounds, driven by increasing number of targeted therapies. Progress is being made in accelerating compounds into advanced stages. However, fluctuating trends are being observed in average duration across three phases.

Increased understanding of underlying causes of Friedreich Ataxia condition and increased access to investments is encouraging growth of Friedreich Ataxia drug pipeline. However, despite high levels of pipeline activity, increasing clinical trial costs and decreasing drug approval rates is requiring companies to focus more on understanding clinical development pipeline compounds.

The research study is structured to provide clear and actionable insights into Friedreich Ataxia drug pipeline for companies to optimize spending levels by understanding competition, their pipeline products and prospect of further advances over the next few years.

The report addresses the discovery, research and pre-clinical stage active Friedreich Ataxia therapeutic candidates. Further, drugs in clinical stage including phase 1, phase 2, phase 3 and pre-registration phases are also analyzed.

The current report examines the current Friedreich Ataxia pipeline along with current development status, regulatory progress, mechanism of action, route of administration, collaborating companies, sponsors, molecule type, and research/discovery details.

In addition to drug overview, recent developments, the report also provides mechanism of action, Pre-clinical trial details and Clinical trial details for each of the drug candidates of Friedreich Ataxia. Further, orphan drug status, fast track designation, grants awarded and other special status for Friedreich Ataxia pipeline compounds are also included.
This study was produced independently by VPAResearch from our proprietary databases, primarily to assist users to develop in-depth understanding of 2019 Friedreich Ataxia pipeline and formulate effective research and development strategies.

RESEARCH METHODOLOGY

Our integrated data reports are built through robust research methodology to ensure our users can build on the insights and data accuracy for their expansion plans. Both primary and secondary research methods are used with stringent quality checks at each stage to evaluate trends, analysis and forecasts.

Scope of the Research

Friedreich Ataxia Pipeline candidates-
  • Pre-clinical Phase: Discovery, research, pre-clinical
  • Early Phase: Phase 1 and Phase 2 Friedreich Ataxia drugs
  • Late phase: Phase 3 and in-approval Friedreich Ataxia drugs
Companies involved in the Pipeline-
  • Company overview
  • Snapshot
  • Friedreich Ataxia therapeutic treatment activities
Details for each Friedreich Ataxia drug candidate-
  • Snapshot
    • Drug Name
    • Alternative Names
    • Company
    • Originator
    • Phase
    • Molecule Type
    • Orphan Drug Status
  • Drug Overview
  • Mechanism of Action
  • Current Status
  • Trial Details
Other details
  • Pre-clinical trials for each drug candidate
  • Clinical trials for each drug candidate
Recent Friedreich Ataxia therapeutic candidates, clinical trials, investments, grants, partnerships, licenses, awards and other news
1 TABLE OF CONTENTS

1.1 List of Tables
1.2 List of Figures

2 EXECUTIVE SUMMARY

2.1 Friedreich Ataxia- Disease Overview
2.2 Friedreich Ataxia- Pipeline Snapshot
2.3 Friedreich Ataxia- Pipeline Drugs by Phase
2.4 Friedreich Ataxia- Pipeline Drugs by Company
2.5 Friedreich Ataxia- Pipeline Drugs by Mechanism of Action

3 COMPANY WISE PIPELINE ANALYSIS

3.1 Adverum Biotechnologies Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.2 Agios Pharmaceuticals Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.3 Amylyx Pharmaceutical Corp Friedreich Ataxia Drug Pipeline, H2- 2019
3.4 BioElectron Technology Corp Friedreich Ataxia Drug Pipeline, H2- 2019
3.5 BioMarin Pharmaceutical Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.6 Biovista Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.7 Cardero Therapeutics Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.8 Chondrial Therapeutics LLC Friedreich Ataxia Drug Pipeline, H2- 2019
3.9 Fulcrum Therapeutics Friedreich Ataxia Drug Pipeline, H2- 2019
3.10 Horizon Pharma Plc Friedreich Ataxia Drug Pipeline, H2- 2019
3.11 Ixchel Pharma LLC Friedreich Ataxia Drug Pipeline, H2- 2019
3.12 Jupiter Orphan Therapeutics Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.13 Minoryx Therapeutics SL Friedreich Ataxia Drug Pipeline, H2- 2019
3.14 Panorama Research Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.15 PTC Therapeutics Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.16 Reata Pharmaceuticals Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.17 Retrotope Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.18 STATegics Inc Friedreich Ataxia Drug Pipeline, H2- 2019
3.19 Takeda Pharmaceutical Co Ltd Friedreich Ataxia Drug Pipeline, H2- 2019
3.20 Voyager Therapeutics Inc Friedreich Ataxia Drug Pipeline, H2- 2019

4 PHASE 1 PIPELINE DRUG DETAILS

4.1 Friedreich Ataxia- Phase 1 Drug Details
4.2 Friedreich Ataxia- Phase 1 Drug Overview
4.3 Friedreich Ataxia- Phase 1 Mechanism of Action
4.4 Current Status
4.5 Trial Details

5 PHASE 2 PIPELINE DRUG DETAILS

5.1 Friedreich Ataxia- Phase 2 Drug Details
5.2 Friedreich Ataxia- Phase 2 Drug Overview
5.3 Friedreich Ataxia- Phase 2 Mechanism of Action
5.4 Current Status
5.5 Trial Details

6 PHASE 3 PIPELINE DRUG DETAILS

6.1 Friedreich Ataxia- Phase 3 Drug Details
6.2 Friedreich Ataxia- Phase 3 Drug Overview
6.3 Friedreich Ataxia- Phase 3 Drug Mechanism of Action
6.4 Current Status
6.5 Trial Details

7 PRE-CLINICAL PHASE PIPELINE DRUG DETAILS

7.1 Friedreich Ataxia- Pre-clinical Phase Drug Details
7.2 Friedreich Ataxia- Pre-clinical Phase Drug Overview
7.3 Friedreich Ataxia- Pre-clinical Phase Drug Mechanism of Action
7.4 Current Status
7.5 Trial Details

8 RECENT NEWS AND DEVELOPMENTS

9 APPENDIX

9.1 About VPAResearch
9.2 Sources and Research Methodology


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