Cystic Fibrosis - Pipeline Review - H1, 2014

Published: April 2014 


Global Markets Direct’s, ‘Cystic Fibrosis - Pipeline Review, H1 2014’, provides an overview of the Cystic Fibrosis’s therapeutic pipeline.

This report provides comprehensive information on the therapeutic development for Cystic Fibrosis, complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases. It also reviews key players involved in the therapeutic development for Cystic Fibrosis and special features on late-stage 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, SEC filings, investor presentations and featured press releases from company/university sites and industry-specific third party sources, put together by Global Markets Direct’s team. Drug profiles/records featured in the report undergoes periodic updation following a stringent set of processes that ensures that all the profiles are updated with the latest set of information. Additionally, processes including live news & deals tracking, browser based alert-box and clinical trials registries tracking ensure that the most recent developments are captured on a real time basis.

The report enhances decision making capabilities and help to create effective counter strategies to gain competitive advantage. It strengthens R&D pipelines by identifying new targets and MOAs to produce first-in-class and best-in-class products.

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



  • The report provides a snapshot of the global therapeutic landscape of Cystic Fibrosis
  • The report reviews key pipeline products under drug profile section which includes, product description, MoA and R&D brief, licensing and collaboration details & other developmental activities
  • The report reviews key players involved in the therapeutics development for Cystic Fibrosis and enlists all their major and minor projects
  • The report summarizes all the dormant and discontinued pipeline projects
  • A review of the Cystic Fibrosis products under development by companies and universities/research institutes based on information derived from company and industry-specific sources
  • Pipeline products coverage based on various stages of development ranging from pre-registration till discovery and undisclosed stages
  • A detailed assessment of monotherapy and combination therapy pipeline projects
  • Coverage of the Cystic Fibrosis pipeline on the basis of target, MoA, route of administration and molecule type
  • Latest news and deals relating related to pipeline products

Reasons to Buy

  • Provides strategically significant competitor information, analysis, and insights to formulate effective R&D development strategies
  • Identify emerging players with potentially strong product portfolio and create effective counter-strategies to gain competitive advantage
  • Develop strategic initiatives by understanding the focus areas of leading companies
  • Identify and understand important and diverse types of therapeutics under development for Cystic Fibrosis
  • Plan mergers and acquisitions effectively by identifying key players of the most promising pipeline
  • Devise corrective measures for pipeline projects by understanding Cystic Fibrosis 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
Table of Contents
Table of Contents
Table of Contents 2
List of Tables 6
List of Figures 8
Introduction 9
Global Markets Direct Report Coverage 9
Cystic Fibrosis Overview 10
Therapeutics Development 11
Pipeline Products for Cystic Fibrosis - Overview 11
Pipeline Products for Cystic Fibrosis - Comparative Analysis 12
Cystic Fibrosis - Therapeutics under Development by Companies 13
Cystic Fibrosis - Therapeutics under Investigation by Universities/Institutes 18
Cystic Fibrosis - Pipeline Products Glance 20
Late Stage Products 20
Clinical Stage Products 21
Early Stage Products 22
Unknown Stage Products 23
Cystic Fibrosis - Products under Development by Companies 24
Cystic Fibrosis - Products under Investigation by Universities/Institutes 29
Cystic Fibrosis - Companies Involved in Therapeutics Development 30
Genzyme Corporation 30
Johnson & Johnson 31
GlaxoSmithKline plc 32
Gilead Sciences, Inc. 33
Grifols, S.A. 34
Vectura Group plc 35
Novartis AG 36
Chiesi Farmaceutici SpA 37
Cubist Pharmaceuticals, Inc. 38
Pfizer Inc. 39
Vertex Pharmaceuticals Incorporated 40
PARI Pharma GmbH 41
Harbor Therapeutics, Inc. 42
Pharmaxis Limited 43
Protalix BioTherapeutics, Inc. 44
Xenetic Biosciences plc 45
Galapagos NV 46
Kamada Ltd. 47
Novabiotics Ltd 48
Nostrum Pharmaceuticals, LLC 49
Pulmatrix, Inc. 50
PTC Therapeutics, Inc. 51
Polyphor Ltd. 52
Concert Pharmaceuticals, Inc. 53
NanoBio Corporation 54
Invion Limited 55
Celsus Therapeutics Plc 56
Northern Therapeutics, Inc. 57
DiscoveryBiomed, Inc. 58
Progenra, Inc. 59
Beech Tree Labs, Inc. 60
N30 Pharmaceuticals 61
Errant Gene Therapeutics, LLC 62
AlgiPharma AS 63
Celtaxsys, Inc. 64
Lamellar Biomedical Ltd 65
Microbion Corporation 66
Alaxia SAS 67
ReveraGen BioPharma, Inc. 68
Curaxys, S.L. 69
Carolus Therapeutics, Inc. 70
InvivoGen Therapeutics 71
Serendex Pharmaceuticals A/S 72
ProQR Therapeutics BV 73
JHL Biotech, Inc. 74
Mucokinetica Ltd. 75
Ockham Biotech Limited 76
Cystic Fibrosis - Therapeutics Assessment 77
Assessment by Monotherapy Products 77
Assessment by Combination Products 78
Assessment by Target 79
Assessment by Mechanism of Action 84
Assessment by Route of Administration 89
Assessment by Molecule Type 92
Drug Profiles 95
mannitol - Drug Profile 95
ataluren - Drug Profile 97
lumacaftor - Drug Profile 100
VR-496 - Drug Profile 102
mannitol - Drug Profile 103
Gene Therapy to Activate CFTR for Cystic Fibrosis - Drug Profile 105
digitoxin_ - Drug Profile 106
OligoG For Cystic Fibrosis - Drug Profile 107
alpha-1-proteinase inhibitor (human) - Drug Profile 109
BTL-na - Drug Profile 113
Recombinant A1PI - Drug Profile 114
VX-661 - Drug Profile 115
(fosfomycin + tobramycin) - Drug Profile 116
nitric oxide - Drug Profile 117
N-6022 - Drug Profile 118
QBW-251 - Drug Profile 120
PUR-118 - Drug Profile 121
CTX-4430 - Drug Profile 122
cysteamine - Drug Profile 123
PulmoXen - Drug Profile 124
N-91115 - Drug Profile 125
GS-5737 - Drug Profile 126
Cystic Fibrosis Phage - Drug Profile 127
Small Molecule for Respiratory Disorders - Drug Profile 128
sargramostim - Drug Profile 129
Heparin - Drug Profile 130
HE-3286 - Drug Profile 132
POL-6014 - Drug Profile 134
nadolol - Drug Profile 136
Small Molecules to Activate CFTR for Cystic Fibrosis - Drug Profile 138
solithromycin - Drug Profile 139
CFX-1 - Drug Profile 141
ALX-009 - Drug Profile 142
VBP-15 - Drug Profile 143
CT-2009 - Drug Profile 145
PUR-0400 - Drug Profile 146
ORP-100 - Drug Profile 147
dapsone - Drug Profile 148
NB-401 - Drug Profile 149
N-91138 - Drug Profile 150
1D-11 - Drug Profile 151
Cell Based Gene Therapy For Cystic Fibrosis - Drug Profile 152
Pseudomonas aeruginosa Vaccine - Drug Profile 153
N-91169 - Drug Profile 154
Recombinant Alpha 1-Antitrypsin biosimilar - Drug Profile 155
GSK-2225745 - Drug Profile 156
RV-1088 - Drug Profile 157
Anti-Sense Oligonucleotide for Cystic Fibrosis - Drug Profile 158
Recombinant Human Follistatin - Drug Profile 159
D-Ivacaftor - Drug Profile 160
PRX-110 - Drug Profile 161
Recombinant Human Surfactant Protein D - Drug Profile 162
LMS-611 - Drug Profile 163
Recombinant Human Acid Ceramidase - Drug Profile 164
FDL-169 - Drug Profile 165
Drug For Cystic Fibrosis - Drug Profile 166
GLPG-1837 - Drug Profile 167
fosfomycin - Drug Profile 169
fenretinide - Drug Profile 170
Drugs for COPD and Cystic Fibrosis - Drug Profile 171
Drug to Modulate Kir 4.2 - Drug Profile 172
Enzyme Targeting DNA for Cystic Fibrosis - Drug Profile 173
N-6547 - Drug Profile 174
Plasmid Gene Therapy for Cystic Fibrosis - Drug Profile 175
Small Molecules to Inhibit Neutrophil Elastases Cystic Fibrosis and CNS Diseases - Drug Profile 176
Drug to inhibit HDAC for Cystic Fibrosis and COPD - Drug Profile 177
VRT-325 - Drug Profile 178
Small Molecule for Pneumonia, COPD, Cystic Fibrosis and Pulmonary Tuberculosis - Drug Profile 179
Tar-1 - Drug Profile 180
Small Molecule to Inhibit EnaC for Cystic Fibrosis - Drug Profile 181
Anti-Microbial Drug for Cystic Fibrosis - Drug Profile 182
Small Molecule for Cystic Fibrosis - Drug Profile 183
PUR-1000 - Drug Profile 184
Small Molecules for Bacterial Infections, Oncology and Cystic Fibrosis - Drug Profile 185
PN-1 - Drug Profile 186
SP-9 - Drug Profile 187
SP-14 - Drug Profile 188
MKA-104 - Drug Profile 189
dornase alfa biosimilar - Drug Profile 190
Small Molecules for Cystic Fibrosis - Drug Profile 191
Bifunctional Antibiotics for Anthrax and Cystic Fibrosis - Drug Profile 192
Small Molecules to Inhibit USP8 for Cystic Fibrosis - Drug Profile 193
Next Generation Correctors - Drug Profile 194
nitric oxide - Drug Profile 195
Cystic Fibrosis - Recent Pipeline Updates 196
Cystic Fibrosis - Dormant Projects 227
Cystic Fibrosis - Discontinued Products 232
Cystic Fibrosis - Product Development Milestones 233
Featured News & Press Releases 233
Appendix 242
Methodology 242
Coverage 242
Secondary Research 242
Primary Research 242
Expert Panel Validation 242
Contact Us 243
Disclaimer 243