Fabry Disease – Pipeline Review, H2 2018

Global Markets Direct’s, ‘Fabry Disease – Pipeline Review, H2 2018’, provides an overview of the Fabry Disease pipeline landscape.

The report provides comprehensive information on the therapeutics under development for Fabry Disease, complete with analysis by stage of development, drug target, mechanism of action (MoA), route of administration (RoA) and molecule type. The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases. Additionally, the report provides an overview of key players involved in therapeutic development for Fabry Disease and features dormant and discontinued projects.

Global Markets Direct’s report features investigational drugs from across globe covering over 20 therapy areas and nearly 3,000 indications. The report is built using data and information sourced from Global Markets Direct’s proprietary databases, company/university websites, clinical trial registries, conferences, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources. Drug profiles featured in the report undergoes periodic review following a stringent set of processes to ensure that all the profiles are updated with the latest set of information. Additionally, various dynamic tracking processes ensure that the most recent developments are captured on a real time basis.

The report helps in identifying and tracking emerging players in the market and their portfolios, enhances decision making capabilities and helps to create effective counter strategies to gain competitive advantage.

Note*: Certain sections in the report may be removed or altered based on the availability and relevance of data.

Scope

– The report provides a snapshot of the global therapeutic landscape of Fabry Disease

– The report reviews pipeline therapeutics for Fabry Disease by companies and universities/research institutes based on information derived from company and industry-specific sources

– The report covers pipeline products based on various stages of development ranging from pre-registration till discovery and undisclosed stages

– The report features descriptive drug profiles for the pipeline products which includes, product description, descriptive MoA, R&D brief, licensing and collaboration details & other developmental activities

– The report reviews key players involved Fabry Disease therapeutics and enlists all their major and minor projects

– The report assesses Fabry Disease therapeutics based on drug target, mechanism of action (MoA), route of administration (RoA) and molecule type

– The report summarizes all the dormant and discontinued pipeline projects

– The report reviews latest news related to pipeline therapeutics for Fabry Disease

Reasons to buy

- Gain strategically significant competitor information, analysis, and insights to formulate effective R&D strategies

- Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage

- Identify and understand important and diverse types of therapeutics under development for Fabry Disease

- Identify potential new clients or partners in the target demographic

- Develop strategic initiatives by understanding the focus areas of leading companies

- Plan mergers and acquisitions effectively by identifying key players and it’s most promising pipeline therapeutics

- Devise corrective measures for pipeline projects by understanding Fabry Disease pipeline depth and focus of Indication therapeutics

- Develop and design in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope

- Modify the therapeutic portfolio by identifying discontinued projects and understanding the factors that drove them from pipeline

Companies mentioned

Amicus Therapeutics Inc

Avrobio Inc

Biosidus SA

Chiesi Farmaceutici SpA

Genzyme Corp

greenovation Biotech GmbH

iBio Inc

Idorsia Pharmaceutical Ltd

JCR Pharmaceuticals Co Ltd

Moderna Therapeutics Inc

Pharming Group NV

Resverlogix Corp

Sangamo Therapeutics Inc

Table of Contents

Table of Contents

List of Tables

List of Figures

Introduction

Global Markets Direct Report Coverage

Fabry Disease Overview

Fabry Disease Therapeutics Development

Pipeline Overview

Pipeline by Companies

Pipeline by Universities/Institutes

Products under Development by Companies

Products under Development by Universities/Institutes

Fabry Disease Therapeutics Assessment

Assessment by Target

Assessment by Mechanism of Action

Assessment by Route of Administration

Assessment by Molecule Type

Fabry Disease Companies Involved in Therapeutics Development

Amicus Therapeutics Inc

Avrobio Inc

Biosidus SA

Chiesi Farmaceutici SpA

Genzyme Corp

greenovation Biotech GmbH

iBio Inc

Idorsia Pharmaceutical Ltd

JCR Pharmaceuticals Co Ltd

Moderna Therapeutics Inc

Pharming Group NV

Resverlogix Corp

Sangamo Therapeutics Inc

Fabry Disease Drug Profiles

(ATB-101 + migalastat hydrochloride) Drug Profile

Product Description

Mechanism Of Action

R&D Progress

agalsidase alfa Drug Profile

Product Description

Mechanism Of Action

R&D Progress

agalsidase beta biosimilar Drug Profile

Product Description

Mechanism Of Action

R&D Progress

apabetalone Drug Profile

Product Description

Mechanism Of Action

R&D Progress

AVRRD-01 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate Alpha Galactosidase A for Fabry Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Gene Therapy to Activate GLA for Fabry Disease Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ibiglustat Drug Profile

Product Description

Mechanism Of Action

R&D Progress

lucerastat Drug Profile

Product Description

Mechanism Of Action

R&D Progress

migalastat hydrochloride Drug Profile

Product Description

Mechanism Of Action

R&D Progress

MOSS-AGAL Drug Profile

Product Description

Mechanism Of Action

R&D Progress

mRNA-3630 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

pegunigalsidase alfa Drug Profile

Product Description

Mechanism Of Action

R&D Progress

PGN-005 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

ST-920 Drug Profile

Product Description

Mechanism Of Action

R&D Progress

Fabry Disease Dormant Projects

Fabry Disease Discontinued Products

Fabry Disease Product Development Milestones

Featured News & Press Releases

Aug 10, 2018: FDA approves Galafold (migalastat) for the treatment of certain adult patients with Fabry Disease

May 30, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Fabry Disease in Japan

May 25, 2018: Protalix BioTherapeutics Announces Poster Presentation on Baseline Characteristics for Fabry Disease Patients Screened in the Phase III BALANCE Study of Pegunigalsidase Alfa at the 55th ERA-EDTA Congress

May 16, 2018: Idorsia initiates MODIFY, a Phase 3 registration study to assess lucerastat as a potential new treatment option for patients with Fabry disease

Mar 22, 2018: Amicus Therapeutics Announces Approval of Galafold (Migalastat) for Fabry Disease in Japan

Feb 12, 2018: U.S. FDA Files New Drug Application Under Priority Review for Migalastat for Treatment of Fabry Disease

Jan 31, 2018: Protalix BioTherapeutics' pegunigalsidase alfa Receives Fast Track Designation from the U.S. Food and Drug Administration

Jan 29, 2018: JCR to Present Data On JR-051 at the 14th Annual WORLD Symposium 2018

Jan 29, 2018: Canadian Drug Expert Committee Grants Positive Recommendation for Reimbursement of Galafold (migalastat) for Fabry Disease in Canada

Jan 29, 2018: Protalix BioTherapeutics to Participate in the 14th Annual WORLDSymposium 2018

Jan 17, 2018: Amicus Therapeutics Launches Galafold (Migalastat) for Treatment of Fabry Disease in Spain

Dec 27, 2017: Protalix BioTherapeutics’ PRX-102 Granted Orphan Drug Designation by the European Commission

Dec 19, 2017: JCR Signs MoU with the Grand Duchy of Luxembourg on the Leasing of Industrial Site

Dec 14, 2017: Amicus Therapeutics Submits New Drug Application to U.S. FDA for Migalastat for Treatment of Fabry Disease

Nov 27, 2017: Protalix BioTherapeutics Presents Positive Results from the Phase I/II Open-Label Extension Trial for PRX-102 at the New Horizons for Fabry Disease Conference

Appendix

Methodology

Coverage

Secondary Research

Primary Research

Expert Panel Validation

Contact Us

Disclaimer

List of Tables

List of Tables

Number of Products under Development for Fabry Disease, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products under Development by Universities/Institutes, H2 2018

Products under Development by Companies, H2 2018

Products under Development by Universities/Institutes, H2 2018

Number of Products by Stage and Target, H2 2018

Number of Products by Stage and Mechanism of Action, H2 2018

Number of Products by Stage and Route of Administration, H2 2018

Number of Products by Stage and Molecule Type, H2 2018

Fabry Disease Pipeline by Amicus Therapeutics Inc, H2 2018

Fabry Disease Pipeline by Avrobio Inc, H2 2018

Fabry Disease Pipeline by Biosidus SA, H2 2018

Fabry Disease Pipeline by Chiesi Farmaceutici SpA, H2 2018

Fabry Disease Pipeline by Genzyme Corp, H2 2018

Fabry Disease Pipeline by greenovation Biotech GmbH, H2 2018

Fabry Disease Pipeline by iBio Inc, H2 2018

Fabry Disease Pipeline by Idorsia Pharmaceutical Ltd, H2 2018

Fabry Disease Pipeline by JCR Pharmaceuticals Co Ltd, H2 2018

Fabry Disease Pipeline by Moderna Therapeutics Inc, H2 2018

Fabry Disease Pipeline by Pharming Group NV, H2 2018

Fabry Disease Pipeline by Resverlogix Corp, H2 2018

Fabry Disease Pipeline by Sangamo Therapeutics Inc, H2 2018

Fabry Disease Dormant Projects, H2 2018

Fabry Disease Discontinued Products, H2 2018

List of Figures

List of Figures

Number of Products under Development for Fabry Disease, H2 2018

Number of Products under Development by Companies, H2 2018

Number of Products by Targets, H2 2018

Number of Products by Stage and Targets, H2 2018

Number of Products by Mechanism of Actions, H2 2018

Number of Products by Stage and Mechanism of Actions, H2 2018

Number of Products by Routes of Administration, H2 2018

Number of Products by Stage and Routes of Administration, H2 2018

Number of Products by Molecule Types, H2 2018

Number of Products by Stage and Molecule Types, H2 2018

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