Global Cystic Fibrosis Strategic Business Report 2017 – Research and Markets

Global Cystic Fibrosis Strategic Business Report 2017 – Research and Markets

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Research and Markets has announced the addition of the “Cystic Fibrosis – Global Strategic Business Report” report to their offering.

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This report analyzes the worldwide markets for Cystic Fibrosis in US$ Million. The report provides separate comprehensive analytics for the US, Canada, Europe, and Rest of World. Annual estimates and forecasts are provided for the period 2014 through 2022. Market data and analytics are derived from primary and secondary research.

The report profiles 56 companies including many key and niche players such as

  • Advanced Inhalation Therapies (AIT) Ltd (Israel)
  • Alaxia SAS (France)
  • AlgiPharma AS (Norway)
  • Anthera Pharmaceuticals, Inc. (USA)
  • Arch BioPartners (Canada)
  • Celtaxsys, Inc. (USA)
  • Concert Pharmaceuticals, Inc. (USA)
  • Corbus Pharmaceuticals Holdings, Inc.(USA)
  • CURx Pharmaceuticals (USA)
  • Nivalis Therapeutics, Inc. (USA)
  • NovaBiotics Ltd (UK)
  • Parion Sciences, Inc. (USA)
  • Pharmaxis Ltd (Australia)
  • PTC Therapeutics, Inc. (USA)
  • Pulmatrix, Inc. (USA)
  • AbbVie Inc. (USA)
  • Galapagos NV (Belgium)
  • Genentech, Inc. (USA)
  • Gilead Sciences, Inc. (USA)
  • Novartis AG (Switzerland)
  • Vertex Pharmaceuticals (USA)

Key Topics Covered:

1. MARKET OVERVIEW
Cystic Fibrosis
A Prelude
CF Disease Prevalence: A Brief Note
Treatment Modalities for Cystic Fibrosis
CFTR Modulators: Key Drug Category
Competitive Scenario

2. RESEARCH IN CYSTIC FIBROSIS SPACE
Areas of Research & Development
Gene Therapy
A Step Towards Cure
Research Findings & Innovations
Updated CF Diagnosis Guidelines to Aid in Diagnosis and Personalized Treatment
Pipeline Drugs
An Overview
Select Pipeline Drugs for Cystic Fibrosis Treatment by Development Stage, 2016
Description of Select Pipeline Drugs
Ataluren
VX-661 + Ivacaftor
Riociguat
QBW251
CTP-656
Cavosonstat (N91115)
VX-440
QR-010
pGM169/GL67A
Acebilustat
Resunab
Inhaled Mannitol
VX-371
Inhaled Levoflaxacin (Quinsair)
AeroVanc (Vancomycin Inhalation Powder)

3. DISEASE OVERVIEW
Cystic Fibrosis
A Hereditary Disease
Mutation in CFTR Gene
A Major Cause of CF
?F508
The Most Common Mutation that Affects CFTR Protein
Hereditary Risk of the Disease
Pathophysiology of the Disease
Symptoms of Cystic Fibrosis
Common Symptoms in Early Childhood
Common Symptoms in Grownups or Adults:
Complications Associated with Cystic Fibrosis
Sinus and Lung Illness
Pancreatic, Gastrointestinal, and Liver Disease
Endocrine Disorders
Infertility
Diagnosis of Cystic Fibrosis
Newborn Test
Sweat Test
The IRT Test
Carrier Test
Antenatal Test
Other Common Tests
Prenatal Diagnosis of Cystic Fibrosis
Tests to Diagnose CF-Related Complications
Cystic Fibrosis in Children

4. CYSTIC FIBROSIS TREATMENT: AN OVERVIEW
Existing Treatment Options for Cystic Fibrosis
A Quick Glance at Common Drugs for Cystic Fibrosis Management
Analysis of Drugs Indicated for Management of Cystic Fibrosis
Antibiotic Therapy for Lung Infections
Select Antibiotics Approved for CF Treatment
TOBI (Tobramycin Inhalation Solution)
Other Common Cystic Fibrosis Infections and Recommended Antibiotics
Cayston (Aztreonam for Inhalation Solution)
Combination Antibiotic Therapy
Mucolytics
Pulmozyme
The Effective Mucolytic Agent
Pancreatic Enzyme Replacement Therapy (PERT) for EPI
Leading PE Drugs Worldwide for Treating Pancreatic Insufficiency Associated with Cystic Fibrosis
CTFR Modulators
Bronchodilators
Additional Treatment for Patients with CF
Surgical and Other Techniques
Lung Transplantation
Recommended for Rare Cases of CF
Feeding Tube
For Extra Nutrition
Bowel Surgery
To Remove any Blockage in Bowel

5. CLINICAL STUDIES
Vertex Pharmaceuticals Announces Positive Phase 3 Study Results of ORKAMBI
ProQR Therapeutics Announces Positive Proof-of-Concept Study for QR-010
Concert Pharmaceuticals Presents Positive Food Effect Results for CTP-656
Vertex Pharmaceuticals Initiates Phase 2 Studies for VX-440 & VX-152
Vertex Pharmaceuticals Provides Update on Phase 3 Study of VX
661 with ivacaftor
Protalix BioTherapeutics Announces Start of Phase II Trial of AIR DNase (PRX-110)
Nivalis Therapeutics Announces Completion of Enrollment for Phase 2 Study of N91115
Alaxia SAS Announces Agreement and Grant from CFFT for ALX-009 Studies
Polyphor Completes Successful Phase I Study of Inhaled POL6014
AlgiPharma Receives Grant from CFFT for Phase 2 Trials of OligoG
Health Canada Grants Clearance to ProMetic for its PBI-4050
Cystic Fibrosis Canada Finances Laurent Pharmaceuticals’ LAU- 7b Phase 2 Trial
Galapagos NV Begins Phase 2 Study of GLPG1837
Pharmaxis Announces Positive Phase II Trial Results of Bronchitol®
PTC Therapeutics Completes Enrollment for second Phase 3 clinical trial of Translarna
Anthera Pharmaceuticals Begins Phase 3 Study of Oral Liprotamase
Parion Sciences Announces Expanded Enrollment Criteria for CLEAN-CF Trial
Laurent Announces Positive Phase 1b Trial Results for LAU-7b
Celtaxsys Announces Positive Phase 1 Clinical Trial Data of CTX-4430
Corbus Pharmaceuticals Receives USFDA Clearance to Initiate Phase 2 Study of Resunab
Savara Pharmaceuticals Unveils Positive Phase 2 Trial Results of AeroVanc
KaloBios Reports Top-line Data from Phase 2 Study of KB001-A
Galapagos NV Nominates GLPG2222 for Combination Therapy with GLPG1837
Cystic Fibrosis Trust and NovaBiotics Partner for the Phase IIa Study of Lynovex
PTC Therapeutics Initiates Phase 3 Trial of Translarna/ataluren
Kamada to Start Clinical Study of Alpha-1 Antitrypsin (AAT) for Cystic Fibrosis

6. PRODUCT LAUNCHES/APPROVALS
EC Grants Orphan Drug Designation to Corbus Pharmaceuticals’ Resunab
FDA Approves Vertex’s ORKAMBI® for Extended Age Group
Pharmaxis Announces Approval of Bronchitol® in Russia
FDA Grants Orphan Drug Status to Pulmatrix’s CF Drug PUR1900
ProQR Therapeutics Announces FDA Fast Track Designation for QR-010
Alcresta Therapeutics Launches Relizorb Cartridge
EC Grants Orphan Medicinal Product Designation to Arch Biopartners’ AB569
TGV-Inhalonix Receives Orphan Drug Designation for its Mul-1867
RespirTech Launches Development of Remote Monitoring System for CF
Vertex Pharmaceuticals Receives TGA Approval of ORKAMBI®
AlgiPharma’s OligoG Receives Orphan Drug Designation by FDA
Proteostasis Therapeutics Receives FDA Fast Track designation for PTI-428
Proteostasis Therapeutics Expands CF Drug Pipeline
Vertex Pharmaceuticals Receives Two EU Approvals for KALYDECO®
USPTO Issues Patent to OrPro Therapeutics’ Theradux
FDA Approves Vertex’s ORKAMBI
Advanced Inhalation Therapies Receives Orphan Medicinal Product Designation for AIT-CF from EMA
FDA Approves New Age Group Indication for Vertex Pharmaceuticals’ KALYDECO
Sandoz Launches Tobramycin Inhalation Solution in the US
FDA Approves sNDA for KALYDECO
EC Grants Orphan Designation to Macrolide CF Drug by Synovo GmbH

7. RECENT INDUSTRY ACTIVITY
Pharmaxis Enters into Research Collaboration with Woolcock Institute
Horizon Pharma Acquires Raptor Pharmaceutical
AstraZeneca to Sell Drug Commercialization Rights to Pfizer
Vertex Pharmaceuticals and Moderna Therapeutics Enter into Research Collaboration
Editas Medicine Enters into Agreement with CFFT for CRISPR/ Cas9-based CF Drugs
Galapagos and AbbVie Expand Agreement for CF Portfolio
Raptor Pharmaceutical Acquires Quinsair from Tripex Pharmaceuticals
Galapagos NV Enters into Licensing Agreement with the HUB Foundation
Zambon SpA Acquires Nigaard Pharma
ManRos and Cyclacel Enter into Licensing Agreement for Seliciclib
Vertex and Parion Sciences Collaborate to Develop Investigational CF Treatment
Ruthigen and Pulmatrix Enter into Merger Agreement
N30 Pharmaceuticals Changes Name to Nivalis Therapeutics
Anthera Pharmaceuticals Acquires Sollpura/liprotamase from Eli Lilly
OPKO Health Acquires Inspiromatic dry powder inhaler from Inspiro Medical
CURx Pharmaceuticals Enters into Global License Agreement with Gilead Sciences

8. SELECT CLINICAL-STAGE PLAYERS
Advanced Inhalation Therapies (AIT) Ltd (Israel)
Alaxia SAS (France)
AlgiPharma AS (Norway)
Anthera Pharmaceuticals, Inc. (USA)
Arch BioPartners (Canada)
Celtaxsys, Inc. (USA)
Concert Pharmaceuticals, Inc. (USA)
Corbus Pharmaceuticals Holdings, Inc.(USA)
CURx Pharmaceuticals (USA)
Nivalis Therapeutics, Inc. (USA)
NovaBiotics Ltd (UK)
Parion Sciences, Inc. (USA)
Pharmaxis Ltd (Australia)
PTC Therapeutics, Inc. (USA)
Pulmatrix, Inc. (USA)

9. FOCUS ON SELECT GLOBAL PLAYERS
AbbVie Inc. (USA)
Galapagos NV (Belgium)
Genentech, Inc. (USA)
Gilead Sciences, Inc. (USA)
Novartis AG (Switzerland)
Vertex Pharmaceuticals (USA)

10. GLOBAL MARKET PERSPECTIVE

Total Companies Profiled: 56 (including Divisions/Subsidiaries 58)

The United States (30)
Canada (4)
Europe (20)
France (2)
Germany (2)
– The United Kingdom (3)
Italy (2)
– Rest of Europe (11)
Asia-Pacific (Excluding Japan) (1)
Middle East (3)

For more information about this report visit http://www.researchandmarkets.com/research/v7p7vg/cystic_fibrosis

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Research and Markets
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