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PRN: Global Cystic Fibrosis Strategic Business Report 2017 – Research and Markets






DUBLIN, Mar 20, 2017 /PRNewswire/ —
Research and Markets has announced the addition of the “Cystic Fibrosis – Global Strategic Business Report” report to their offering.

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 OVERVIEWCystic FibrosisA PreludeCF Disease Prevalence: A Brief NoteTreatment Modalities for Cystic FibrosisCFTR Modulators: Key Drug CategoryCompetitive Scenario
2. RESEARCH IN CYSTIC FIBROSIS SPACEAreas of Research & DevelopmentGene TherapyA Step Towards CureResearch Findings & InnovationsUpdated CF Diagnosis Guidelines to Aid in Diagnosis and Personalized TreatmentPipeline DrugsAn OverviewSelect Pipeline Drugs for Cystic Fibrosis Treatment by Development Stage, 2016Description of Select Pipeline DrugsAtalurenVX-661 + IvacaftorRiociguatQBW251CTP-656Cavosonstat (N91115)VX-440QR-010pGM169/GL67AAcebilustatResunabInhaled MannitolVX-371Inhaled Levoflaxacin (Quinsair)AeroVanc (Vancomycin Inhalation Powder)
3. DISEASE OVERVIEW !Cystic FibrosisA Hereditary DiseaseMutation in CFTR GeneA Major Cause of CF?F508The Most Common Mutation that Affects CFTR ProteinHereditary Risk of the DiseasePathophysiology of the DiseaseSymptoms of Cystic FibrosisCommon Symptoms in Early ChildhoodCommon Symptoms in Grownups or Adults:Complications Associated with Cystic FibrosisSinus and Lung IllnessPancreatic, Gastrointestinal, and Liver DiseaseEndocrine DisordersInfertilityDiagnosis of Cystic FibrosisNewborn TestSweat TestThe IRT TestCarrier TestAntenatal TestOther Common TestsPrenatal Diagnosis of Cystic FibrosisTests to Diagnose CF-Related ComplicationsCystic Fibrosis in Children
4. CYSTIC FIBROSIS TREATMENT: AN OVERVIEWExisting Treatment Options for Cystic FibrosisA Quick Glance at Common Drugs for Cystic Fibrosis ManagementAnalysis of! Drugs Indicated for Management of Cystic FibrosisAntibi! otic The rapy for Lung InfectionsSelect Antibiotics Approved for CF TreatmentTOBI (Tobramycin Inhalation Solution)Other Common Cystic Fibrosis Infections and Recommended AntibioticsCayston (Aztreonam for Inhalation Solution)Combination Antibiotic TherapyMucolyticsPulmozymeThe Effective Mucolytic AgentPancreatic Enzyme Replacement Therapy (PERT) for EPILeading PE Drugs Worldwide for Treating Pancreatic Insufficiency Associated with Cystic FibrosisCTFR ModulatorsBronchodilatorsAdditional Treatment for Patients with CFSurgical and Other TechniquesLung TransplantationRecommended for Rare Cases of CFFeeding TubeFor Extra NutritionBowel SurgeryTo Remove any Blockage in Bowel
5. CLINICAL STUDIESVertex Pharmaceuticals Announces Positive Phase 3 Study Results of ORKAMBIProQR Therapeutics Announces Positive Proof-of-Concept Study for QR-010Concert ! Pharmaceuticals Presents Positive Food Effect Results for CTP-656Vertex Pharmaceuticals Initiates Phase 2 Studies for VX-440 & VX-152Vertex Pharmaceuticals Provides Update on Phase 3 Study of VX661 with ivacaftorProtalix BioTherapeutics Announces Start of Phase II Trial of AIR DNase (PRX-110)Nivalis Therapeutics Announces Completion of Enrollment for Phase 2 Study of N91115Alaxia SAS Announces Agreement and Grant from CFFT for ALX-009 StudiesPolyphor Completes Successful Phase I Study of Inhaled POL6014AlgiPharma Receives Grant from CFFT for Phase 2 Trials of OligoGHealth Canada Grants Clearance to ProMetic for its PBI-4050Cystic Fibrosis Canada Finances Laurent Pharmaceuticals’ LAU- 7b Phase 2 TrialGalapagos NV Begins Phase 2 Study of GLPG1837Pharmaxis 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 Lip! rotamaseParion Sciences Announces Expanded Enrollment Criteria for CLEAN-CF TrialLaurent Announces Positive Phase 1b Trial Results for LAU-7bCeltaxsys Announces Positive Phase 1 Clinical Trial Data of CTX-4430Corbus Pharmaceuticals Receives USFDA Clearance to Initiate Phase 2 Study of ResunabSavara Pharmaceuticals Unveils Positive Phase 2 Trial Results of AeroVancKaloBios Reports Top-line Data from Phase 2 Study of KB001-AGalapagos NV Nominates GLPG2222 for Combination Therapy with GLPG1837Cystic Fibrosis Trust and NovaBiotics Partner for the Phase IIa Study of LynovexPTC Therapeutics Initiates Phase 3 Trial of Translarna/atalurenKamada to Start Clinical Study of Alpha-1 Antitrypsin (AAT) for Cystic Fibrosis
6. PRODUCT LAUNCHES/APPROVALSEC Grants Orphan Drug Designation to Corbus Pharmaceuticals’ ResunabFDA Approves Vertex’s ORKAMBI® for Extended Age GroupPharmaxis Announces Approval of Bronchit! ol® in RussiaFDA Grants Orphan Drug Status to Pulmatrix’s CF Drug PUR1900ProQR Therapeutics Announces FDA Fast Track Designation for QR-010Alcresta Therapeutics Launches Relizorb CartridgeEC Grants Orphan Medicinal Product Designation to Arch Biopartners’ AB569TGV-Inhalonix Receives Orphan Drug Designation for its Mul-1867RespirTech Launches Development of Remote Monitoring System for CFVertex Pharmaceuticals Receives TGA Approval of ORKAMBI®AlgiPharma’s OligoG Receives Orphan Drug Designation by FDAProteostasis Therapeutics Receives FDA Fast Track designation for PTI-428Proteostasis Therapeutics Expands CF Drug PipelineVertex Pharmaceuticals Receives Two EU Approvals for KALYDECO®USPTO Issues Patent to OrPro Therapeutics’ TheraduxFDA Approves Vertex’s ORKAMBIAdvanced Inhalation Therapies Receives Orphan Medicinal Product Designation for AIT-CF from EMA FDA Approves New Age Group Indication for Vertex Pharmaceut! icals’ K ALYDECOSandoz Launches Tobramycin Inhalation Solution in the USFDA Approves sNDA for KALYDECOEC Grants Orphan Designation to Macrolide CF Drug by Synovo GmbH
7. RECENT INDUSTRY ACTIVITYPharmaxis Enters into Research Collaboration with Woolcock InstituteHorizon Pharma Acquires Raptor PharmaceuticalAstraZeneca to Sell Drug Commercialization Rights to PfizerVertex Pharmaceuticals and Moderna Therapeutics Enter into Research CollaborationEditas Medicine Enters into Agreement with CFFT for CRISPR/ Cas9-based CF DrugsGalapagos and AbbVie Expand Agreement for CF PortfolioRaptor Pharmaceutical Acquires Quinsair from Tripex PharmaceuticalsGalapagos NV Enters into Licensing Agreement with the HUB FoundationZambon SpA Acquires Nigaard PharmaManRos and Cyclacel Enter into Licensing Agreement for SeliciclibVertex and Parion Sciences Collaborate to Develop Investigational CF TreatmentRuthig! en and Pulmatrix Enter into Merger AgreementN30 Pharmaceuticals Changes Name to Nivalis TherapeuticsAnthera Pharmaceuticals Acquires Sollpura/liprotamase from Eli LillyOPKO Health Acquires Inspiromatic dry powder inhaler from Inspiro MedicalCURx Pharmaceuticals Enters into Global License Agreement with Gilead Sciences
8. SELECT CLINICAL-STAGE PLAYERSAdvanced 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 PLAYERSAbbVie 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
Media Contact:
Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.com
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