Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase 3 studies of the tezacaftor/ivacaftor combination treatment, a medicine in development that is designed to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Continue reading Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment

Vertex Drug Gets Priority Review for Cystic Fibrosis

http://www.empr.com/drugs-in-the-pipeline/tezacaftor-ivacaftor-vertex-cystic-fibrosis-priority-review-nda/article/684284/

Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis

The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.

The NDA submission was based on positive results from 2 global Phase 3 trials, which showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in patients treated with tezacaftor/ivacaftor.

The combination treatment consists of ivacaftor (marketed under the brand name Kalydeco), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and tezacaftor, a novel CFTR corrector. Tezacaftor is designed to address the processing defect of F508del-CFTR to enable it to reach the cell surface, where ivacaftor can further enhance the protein’s function.

The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of February 28, 2018 to make a decision on the NDA.

For more information visit Vrtx.com.

Vertex Grants $1 Million to Non-Profits for Cystic Fibrosis

http://www.raredr.com/

Earlier today, Vertex Pharmaceuticals Incorporated – a corporation designed to fund scientific innovation to create transformative medicines for people with serious and life-threatening diseases – announced the grant recipients for its 2017 Cystic Fibrosis (CF) Circle of Care program. Continue reading Vertex Grants $1 Million to Non-Profits for Cystic Fibrosis

New cystic fibrosis drug (Vertex)

https://www.ft.com/content/aa944954-13f4-11e7-b0c1-37e417ee6c76

Combination treatment offers safer option for patients suffering from deadly illness

Vertex Pharmaceuticals said that a combination of two of its medicines had successfully treated cystic fibrosis in a closely watched clinical trial, paving the way for regulators to approve a Continue reading New cystic fibrosis drug (Vertex)

News about Concert Pharmaceuticals’ cystic fibrosis drug

http://cen.acs.org/articles/95/web/2017/03/Vertex-buys-Concerts-cystic-fibrosis-drug.htm

Vertex buys Concert’s cystic fibrosis drug
Vertex Pharmaceuticals will pay up to $250 million for Concert Pharmaceuticals’ cystic fibrosis drug CTP-656. Currently in Phase II clinical trials, the drug is a deuterated version of Vertex’s own Continue reading News about Concert Pharmaceuticals’ cystic fibrosis drug

My Five Takeaways from #NACFC2016

I had a dream last night, and in that dream the Limp Bizkit classic song, Nookie was on repeat for some reason. I have no idea why; I haven’t heard that song in about 12 years. Needless to say I woke up pretty jacked up and motivated to get sh*t done before the Packers game this afternoon. Continue reading My Five Takeaways from #NACFC2016

Vertex ends trial of cystic fibrosis drug combination

https://www.bostonglobe.com/business/2016/08/15/vertex-ends-trial-cystic-fibrosis-drug-combination/6AkHgBOmtKPF9XRgq18IQM/story.html

Vertex Pharmaceuticals Inc. said Monday it is ending a clinical study testing a two-drug combination therapy on a small group of cystic fibrosis patients after an independent board concluded the experimental treatment wasn’t showing Continue reading Vertex ends trial of cystic fibrosis drug combination

Moderna, Vertex to collaborate on cystic fibrosis

https://www.bostonglobe.com/business/2016/07/06/moderna-vertex-collaborate-cystic-fibrosis/rEmPx5Ll746RmQL0oUNIiO/story.html

Moderna Therapeutics Inc. has emerged as a partner of choice for drug makers eager to supplement their own research.

Vertex Pharmaceuticals Inc. became the latest established biopharma company Continue reading Moderna, Vertex to collaborate on cystic fibrosis

Exploring the need for mutation-specific treatments in cystic fibrosis

Shared from: 1 July 2016 Author: Caroline Richards, Editor, European Pharmaceutical Review

Despite the fact that over 1,900 mutations of the cystic fibrosis transmembrane conductance regulator (CFTR) are known to cause cystic fibrosis (CF), a debilitating inherited genetic disorder that progressively reduces organ function Continue reading Exploring the need for mutation-specific treatments in cystic fibrosis

Nivalis Therapeutics Announces First Patient Dosed in Second Phase 2 Study of N91115 for Treatment of Cystic Fibrosis

https://globenewswire.com/news-release/2016/05/16/840427/0/en/Nivalis-Therapeutics-Announces-First-Patient-Dosed-in-Second-Phase-2-Study-of-N91115-for-Treatment-of-Cystic-Fibrosis.html

Nivalis Therapeutics, Inc. (NASDAQ:NVLS), a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (“CF”), today announced the first patient was dosed in a second Phase 2 clinical study of Continue reading Nivalis Therapeutics Announces First Patient Dosed in Second Phase 2 Study of N91115 for Treatment of Cystic Fibrosis