Kalydeco

March 10, 2016March 16, 2016

Nivalis Expands Clinical Development of Its Cystic Fibrosis CFTR Stabilizer

Cystic Fibrosis, News

Nivalis Therapeutics recently announced the expansion of the clinical development plan for N91115, the company’s investigational, first-in-class stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, which is defective in ... Read more

February 10, 2016February 18, 2016

Vertex CF Drug, Kalydeco, Rejected as Therapy for Specific Group of Mutations by FDA

Cystic Fibrosis, News

Vertex Pharmaceuticals recently received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) stating that its supplemental New Drug Application (sNDA) could not be approved in its current ... Read more

November 19, 2015

Vertex’s Cystic Fibrosis Therapy Expands to Younger Children, More Adults

Cystic Fibrosis, News

Vertex Pharmaceuticals Inc. recently confirmed that the European Commission has expanded the indication of Kalydeco® (ivacaftor) to two more groups of patients: children ages 2 to 5 with cystic fibrosis (CF) who have at least ... Read more

November 13, 2015November 13, 2015

Potential New Cystic Fibrosis Therapy, CTP-656, Begins Phase 1 Study

Cystic Fibrosis, News

Concert Pharmaceuticals, Inc., a clinical stage biopharmaceutical company focused on utilizing its DCE Platform® (deuterated chemical entity platform) to create novel small molecule drugs, recently announced that it has initiated a Phase ... Read more

October 12, 2015November 5, 2015

Vertex Presents Key Findings on Kalydeco, Orkambi & Experimental CF Therapies at NACFC

Cystic Fibrosis, News

A series of new updates and future development plans were recently announced by Vertex regarding its approved and candidate drugs to treat Cystic fibrosis (CF). The announcements, made at the 29th Annual ... Read more

September 28, 2015September 28, 2015

CHMP Issues Positive Recommendation for ORKAMBI and KALYDECO in the EU

News

Biotechnology company Vertex Pharmaceuticals, Inc. recently announced that the European Union Committee for Medicinal Products for Human Use (CHMP) has released a positive opinion and recommendation for Marketing Authorization of breakthrough ... Read more

September 23, 2015November 5, 2015

Concert Pharma Reports Phase 1 Results of Experimental CF Therapy CTP-656

Cystic Fibrosis, News

Concert Pharma, a biotechnology company utilizing deuterium chemistry to discover and develop innovative drug products that can address unmet medical needs, recently announced positive results from its Phase 1 single ascending dose study ... Read more

April 14, 2015November 5, 2015

New CF Treatments Could Mean Sky-High Health Costs

Cystic Fibrosis, News

A recent research project by Prime Therapeutics LLC (Prime) found that new treatments targeting specific gene mutations that cause cystic fibrosis could drastically increase costs associated with treating the condition. These results were presented at the Academy of ... Read more

March 27, 2015November 5, 2015

Vertex Shares Slide After Combined Cystic Fibrosis Therapy Disappoints in Preliminary Data

Cystic Fibrosis, News

Shares of Vertex Pharmaceuticals Inc. fell sharply in the wake of disappointing results of the company’s drug combination of experimental drug VX-661 with FDA-approved Kalydeco. The company stated that its investigational cystic fibrosis drug combination drug did ... Read more

March 18, 2015November 5, 2015

Concert Pharma Starts Phase 1 Trial on Deuterated Ivacaftor for CF

Cystic Fibrosis, News

Clinical stage Concert Pharmaceuticals Inc. has initiated a phase 1 clinical study to evaluate a deuterium-modified version of ivacaftor, as a potential therapy for cystic fibrosis (CF). The company is planning on studying ... Read more