Kalydeco Clinical Trials

Breakthrough Therapy Designation - About.com Money
A breakthrough therapy designation gives drug sponsors priority review for their drug, the cystic fibrosis drug Kalydeco (ivacaftor) marketed by Vertex Pharmaceuticals; and Gazyva Making Clinical Trials Data More Transparent; Clinical Trial Basics; ... Read Article

Insights From The Kalydeco Journey: A Real Life Story Of ...
Insights From The Kalydeco Journey: A Real Life Story of Progress and Possibility I n one of the most anticipated presenta-tions of the 2012 National CF Family clinical trials, has held true in clinical practice. Dr. Boyle shared that patients ... Fetch Document

Small Clinical Trials - Food And Drug Administration
Small Clinical Trials Anne Pariser, M.D. Office of Translational Sciences . Center for Drug Evaluation and Research . FDA . September 22, 2014 ... Read Here

Reference ID: 3079771
Because clinical trials are conducted under widely varying conditio ns, Therefore, food containing grapefruit or Seville oranges should be avoided during treatment with KALYDECO [see. Clinical Pharmacology (12.3)]. 7.2 ; Inducers of CYP3A ... Read Document

MilestonesUpdate - Cystic Fibrosis Foundation
Clinical trials of Kalydeco in combination with two other potential drugs in people with two copies of the most common CF mutation, and news that the Foundation has funded new CF projects with Pfizer and Genzyme. These great gains would not be possible without the unwavering ... Access Doc

A Learning Tool About KALYDECO And Fat-containing Foods
Take KALYDECO with fat-containing foods. You may have heard that not all fats are created equal. • During the clinical trials comparing KALYDECO to a sugar pill called placebo, patients took KALYDECO along with their other prescribed CF therapies, except ... View Doc

Www.tga.gov.au
Therapeutic Goods Administration Submission PM-2012-01491-3-5 Extract from the Clinical Evaluation Report for Kalydeco ivacaftor Page 3 of 68 Contents List of abbrevia tions _____ ... Fetch Full Source

Clinical Pharmacology, and “Regulatory Science Public listing of all clinical trials & results Ivacaftor (Kalydeco, cystic fibrosis, Vertex) ... Return Document

Clinical Trials: A Vital Part Of Cystic Fibrosis Treatment ...
Vertex’s Kalydeco was successfully approved after extensive clinical trials in CF patients with G551D mutation. Clinical Trials: A Vital Part of Cystic Fibrosis Treatment Advancements MerAndA Sue. Page 2 CF Roundtable Winter 2014 CF ROUNDTABLE FOUNDED 1990 ... Visit Document

Dean Kalydeco Update - YouTube
Dean Kalydeco Blog 3 part 1 - Duration: 6:43. by DON BOWLER 4,991 views. 6:43 Dean Kalydeco Update!!!!! Cystic Fibrosis Clinical Trials: A Mother Share's Her Daughter's Experience - Duration: 0:36. by Cystic Fibrosis Foundation 1,203 views. ... View Video

PRODUCT MONOGRAPH INCLUDING PATIENT MEDICATION ... - Vrtx.com
The trial design and patient demographics for the KALYDECO clinical trials are summarized in Table 5 below. Table 5 -Trials 1, 2, 3, 4, 5 (KALYDECO Compared with Placebo) and Trial 6 Study Number Trial Design Dosage; Route of Administration; Duration Number of Subjects ... Access Document

Cost-effectiveness Of Ivacaftor (Kalydeco™) For The Treatment ...
Cost-effectiveness of Ivacaftor (Kalydeco™) for the treatment of cystic fibrosis in patients age 6 years and older who have the G551D the primary outcome measure for the two phase III clinical trials. 4. The economic dossier was a cost utility analysis comparing ivacaftor plus standard ... Retrieve Document

Katie Murphy On Patient Involvement In Healthcare Decision ...
Katie Murphy on patient involvement in healthcare decision making HEORtv. Ivacaftor (Kalydeco), developed by Vertex Pharmaceuticals proved effective in treating this type of CF in clinical trials. ... View Video

ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS
Kalydeco should only be prescribed by physicians with experience in the treatment of cystic fibrosis. If the patient's genotype is unknown, clinical trials was similar between subjects in the ivacaftor and placebo treatment groups (see section ... View Full Source

Clinical Trial Trends Among Venture Philanthropy Organizations
Clinical Trial Trends Among Venture Philanthropy Organizations A look at the TRAIN clinical trial Kalydeco Clinical Care Center Network Patients Trials Registry Doctors . Venture philanthropies are making clinical trials more efficient. These organizations are fixing the pipe ... View Doc

Cystic Fibrosis Foundation 2012 Annual Report
Yearlong, international Phase 3 clinical trials of Kalydeco, which showed that people with the G551D mutation Cystic Fibrosis Foundation Condensed Financial Information* Consolidated Statement of Financial Position as of December 31, 2012 and 2011 ... Access Full Source

Temporary Compliance Waiver Notice
Temporary Compliance Waiver Notice Kalydeco for the proposed indication (CF patients 6 years of age and older with a . double-blind, placebo-controlled clinical trials in over 250 CF patients with a G551D mutation in the CFTR was able to be conducted. ... Fetch Doc

Genetic Testing For Cystic Fibrosis - Mutations
Genetic testing for cystic fibrosis is done to determine if a person has, or is a carrier of, cystic fibrosis. The test is usually done on a blood sample, but sometimes can be done on other DNA-containing cells such as a swab from the inside of the cheek. ... Read Article

Chiasma Expands Senior Management Team With Key Strategic Hires
NEWTON, Mass., Jan. 04, 2016-- Chiasma, Inc., a late-stage biopharmaceutical company developing Mycapssa™, an investigational oral drug for the maintenance therapy of adult patients with the orphan disease ... Read News

Drug Development - Wikipedia, The Free Encyclopedia
New chemical entity development. Broadly, the process of drug development can be divided into pre-clinical and clinical work. ... Read Article

KALYDECO Full Prescribing Information - Vrtx.com
HIGHLIGHTS OF PRESCRIBING INFORMATION These highlights do not include all the information needed to use KALYDECO safely and effectively. See full prescribing information for KALYDECO. 6.1 Clinical Trials Experience 7 DRUG INTERACTIONS ... Access Doc

Pharmacogenomics In Rare Diseases: Development Strategy For ...
Responded to Kalydeco In Vitro Group Molecular Phenotype Functional Phenotype clinical trials •First used in 1960s for behavioral research Pharmacogenomics in Rare Diseases: Development Strategy for Ivacaftor as a Therapy for Cystic Fibrosis ... Retrieve Full Source

Reference ID: 3711147 - Food And Drug Administration
Clinical trials of KALYDECO did not include sufficient numbers of patients 65 years of age and over to determine whether they respond differently from younger patients. 8.6 Hepatic Impairment . No dose adjustment is necessary for patients with mild hepatic impairment (Child-Pugh Class A). ... Visit Document

Lumacaftor - Wikipedia, The Free Encyclopedia
Lumacaftor (USAN, codenamed VX-809) is an experimental drug for the treatment of cystic fibrosis being developed by Vertex Pharmaceuticals. Results from a Phase II clinical trial indicate that patients with the most common form of genetic mutation causing cystic fibrosis ... Read Article