Therapies

ORKAMBI™ Is Now FDA Approved!

CF Community, CF News, Research, Therapies

March 19, 2019

ORKAMBI is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 12 years and older who have two copies of the F508del mutation (F508del/F508del) in their CFTR gene. ORKAMBI should not be used in patients other than those who have two...

FDA Panel Recommends Approval of ORKAMBI™ via Vertex

by BEF

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE --- Please read the original press release here --- SOURCE FDA decision expected by July 5, 2015 PDUFA date Approximately 8,500 people with cystic fibrosis in the U.S. have two copies of the F508del mutation and are ages 12 and older BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX)...

Yale scientists use gene editing to correct mutation in cystic fibrosis via Yale News

by BEF

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE Yale researchers successfully corrected the most common mutation in the gene that causes cystic fibrosis, a lethal genetic disorder. The study was published April 27 in Nature Communications. Cystic fibrosis is an inherited, life-threatening disorder that damages the lungs and digestive system. It is most commonly caused...

New antibiotic teixobactin kills drug-resistant superbugs, study says via LA Times

by BEF

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE: LA TIMES Using soil from a grassy field in Maine and a miniaturized diffusion chamber, scientists have cultivated a microbe that could help tame the spread of antibiotic-resistant superbugs. When tricked into growing in a lab, the microbe makes a compound that kills strains of tuberculosis,...

Vertex Submits Application for Combination Drug for People with 2 Copies of F508del Mutation

by BEF

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE - VERTEX OFFICIAL PRESS RELEASE November 5, 2014 Vertex Submits Applications in the U.S. and Europe for Approval of Lumacaftor in Combination with Ivacaftor for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation -U.S. submission includes request for Priority Review; Accelerated Assessment has...

FDA Recommends Approval of KALYDECO for Use in People with CF R117H Mutation

by BEF

CF Community, CF News, Research, Therapies

March 19, 2019

SOURCE: VERTEX PRESS RELEASE FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation BOSTON--(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration's Pulmonary Allergy Drugs...

Median Age Of Survival For Canadians With CF Surpasses 50 via CF News Today

by BEF

CF News, Research, Therapies

March 19, 2019

SOURCE In the 1980s, life expectancy for persons with cystic fibrosis (CF) was just 12 years in the US, and around 20 in Canada. However, the median age of survival for Canadian CF patients continues on an upward trend, and is currently estimated to be 50.9...

UNC Researchers Find New CF Drugs Counteract One Another’s Effectiveness via CF News Today

by BEF

CF News, Research, Therapies

March 19, 2019

SOURCE Findings of new study led by Martina Gentzsch, PhD, at the University of North Carolina School of Medicine and the UNC Marsico Lung Institute in Chapel Hill, N.C. could help drug developers improve compounds designed to correct CFTR proteins in cystic fibrosis (CF) patients. In...

Vertex’s Two-Drug Cystic Fibrosis Treatment Shows Promise in Clinical Trials via NY Times

by BEF

CF News, Research, Therapies

March 19, 2019

Vertex Pharmaceuticals said on Tuesday that a combination of two of its drugs had successfully treated cystic fibrosis in closely watched clinical trials, potentially clearing the way for approval of a new option for nearly half the patients with the genetic disease. The drug combination, when...

Aptalis Pharma Studies Panzytrat in Cystic Fibrosis Patients in Phase 4 Trial via CF News Today

by BEF

CF News, Research, Therapies

March 19, 2019

via Cystic Fibrosis News Today Aptalis Pharma continued to evaluate its pancreatic enzyme products (PEPs) in patients with cystic fibrosis and exocrine pancreatic insufficiency in its Phase 4 clinical trial of Panzytrat® 25,000. Results show that patients taking Panzytrat had better control over their steatorrhea, or...

Therapies

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