F508del Mutation

April 4, 2017April 4, 2017

Tezacaftor-Kalydeco Combo Is Promising CF Treatment, Phase 3 Trials Show

Cystic Fibrosis, News

A combination of tezacaftor and ivacaftor significantly improved lung function in cystic fibrosis (CF) patients 12 years and older who had a F508del mutation, according to two Phase 3 clinical ... Read more

March 24, 2017March 24, 2017

Galapagos Starts Trial of Therapy It Hopes Becomes Part of Triple-Combination CF Treatment

Cystic Fibrosis, News

Galapagos has started a Phase 1 clinical trial of its novel potentiator GLPG3067 for cystic fibrosis (CF). It is the second Phase 1 trial the company is conducting. The other one ... Read more

June 27, 2016June 27, 2016

Cost of Cystic Fibrosis Therapy Orkambi too High for Low Benefit, Say U.K.’s NICE

Cystic Fibrosis, News

The U.K.’s National Institute for Health and Care Excellence (NICE) announced that is does not recommend funding a cystic fibrosis (CF) treatment Orkambi through Vertex Pharmaceuticals at this time. In March, ... Read more

May 24, 2016May 24, 2016

First Patient Dosed in Phase 2 Trial of Potential CF Treatment

Cystic Fibrosis, News

Nivalis Therapeutics, Inc., announced the first dosing of a cystic fibrosis (CF) patient enrolled in its second Phase 2 clinical trial evaluating the drug N91115, a stabilizer of the cystic fibrosis transmembrane conductance regulator ... Read more

May 4, 2016May 4, 2016

Galapagos, AbbVie Expand Collaboration on Cystic Fibrosis Treatments

Cystic Fibrosis, News

Galapagos NV and AbbVie are expanding their collaboration to continue their successful portfolio growth in cystic fibrosis (CF). The two companies are committed to increasing the potential milestones to Galapagos for Phase ... Read more

December 3, 2015December 3, 2015

Cystic Fibrosis Phase 2 Study of CFTR Stabilizer Begins Treatments

Cystic Fibrosis, News

Nivalis Therapeutics, Inc., announced that a Phase 2 clinical study of its lead investigational drug N91115 has begun treating its first patient. Nivalis, a pharmaceutical company focused on cystic fibrosis (CF) treatments, developed N91115 ... Read more

November 3, 2015November 24, 2015

CF Fertility Rates Research Personal for University of Alabama at Birmingham Investigator

Cystic Fibrosis, News

For Sigrid Ladores, Ph.D., a pediatric nurse practitioner and an Assistant Professor at the University of Alabama at Birmingham School of Nursing, investigating reproductive health concerns of adolescents and young ... Read more

September 4, 2015November 5, 2015

Inflammatory TNF-alpha Found To Prevent Degradation of CFTR Protein in Cystic Fibrosis

Cystic Fibrosis, News

In a new study entitled “An unexpected effect of TNF-α on F508del-CFTR maturation and function,” researchers found that a key factor released during inflammatory responses – TNF-alpha – can prevent ... Read more

May 13, 2015November 5, 2015

Vertex’s New CF Drug Combo Receives Positive FDA Recommendation

Cystic Fibrosis, News

The U.S. Food and Drug Administration (FDA) has issued a positive evaluation of ORKAMBI (lumacaftor/ivacaftor) as a recommended treatment for cystic fibrosis (CF) patients 12 years and older with two copies of the F508del mutation ... Read more

November 6, 2014November 5, 2015

Lumacaftor/Ivacaftor Drug Combo for CF Patients With Two Copies Of F508del Mutation Gains U.S., European Approval

Cystic Fibrosis, News

Vertex Pharmaceuticals Incorporated has announced its submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European ... Read more