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FDA Approves Symdeko (tezacaftor/ivacaftor and ivacaftor) to Treat Cystic Fibrosis in People Ages 12 and Older with Certain Mutations in the CFTR Gene

Posted 14 Feb 2018 by Drugs.com

BOSTON--(BUSINESS WIRE) Feb 12, 2018 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved Symdeko (tezacaftor/ivacaftor and ivacaftor) for treating the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator ...

Kalydeco Approval Widened for More Types of Cystic Fibrosis

Posted 18 May 2017 by Drugs.com

THURSDAY, May 18, 2017 – The U.S. Food and Drug Administration says it has expanded approval for the cystic fibrosis drug Kalydeco (ivacaftor) to include 33 mutations of the disease, up from the previous 10 mutations. In a media release, the agency said its decision was based on results of laboratory testing and previous clinical trials. "Many rare cystic fibrosis mutations have such small ...

Cystic Fibrosis Drug Seems OK for Preschoolers: Study

Posted 25 Jan 2016 by Drugs.com

MONDAY, Jan. 25, 2016 – The cystic fibrosis drug ivacaftor appears safe and effective for young children, a drug company-funded study suggests. "This was a small trial, but we are thrilled to see these results," said study leader Jane Davies, from the National Heart and Lung Institute at Imperial College London in England. "Ivacaftor is a potential new treatment to offer children aged 2 years ...

Finding Disease Cures Can Take Up to a Century: Analysis

Posted 24 Sep 2015 by Drugs.com

THURSDAY, Sept. 24, 2015 – A team of scientists has looked back over decades of discovery to conclude that it can take dozens of years, even a century, for cumulative research to lead to a cure for a single disease. The finding is disheartening given the current U.S. government underfunding of the basic science needed to investigate diseases, said a team led by Dr. R. Sanders Williams, president ...

Orkambi Approved for Cystic Fibrosis

Posted 2 Jul 2015 by Drugs.com

THURSDAY, July 2, 2015 – Orkambi (lumacaftor and ivacaftor) has been approved by the U.S. Food and Drug Administration to treat cystic fibrosis in patients who have inherited two copies of a specific gene mutation from their parents. The F508del mutation causes production of an abnormal protein that disrupts transport of water and chloride through the body, the FDA said Thursday in a news ...

Combination Treatment for Cystic Fibrosis Shows Promise

Posted 18 May 2015 by Drugs.com

SUNDAY, May 17, 2015 – Combining two medications that target the most common genetic cause of cystic fibrosis improved lung function in people with the disease. The combination therapy also reduced the rate of lung infections and other problems, according to a new study. The two drugs are called ivacaftor and lumacaftor. Ivacaftor is already approved by the U.S. Food and Drug Administration for ...

FDA Approves Kalydeco (ivacaftor) for Cystic Fibrosis in Children Ages 2 to 5 with CFTR Gene Mutations

Posted 23 Mar 2015 by Drugs.com

BOSTON, Mar 18, 2015 --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved Kalydeco® for use in children ages 2 to 5 with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, ...

FDA Approves Kalydeco (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation

Posted 30 Dec 2014 by Drugs.com

BOSTON, December 29, 2014 --(BUSINESS WIRE)-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved a supplemental new drug application (sNDA) for the use of Kalydeco (ivacaftor) in people with cystic fibrosis (CF) ages 6 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) ...

Cystic Fibrosis Drug Combo May Be Less Effective Than Hoped

Posted 23 Jul 2014 by Drugs.com

WEDNESDAY, July 23, 2014 – A powerful drug combo may not be as effective against cystic fibrosis as previously thought. New lab research on human cells suggests that one of the medications might stop the other from working properly. However, this study's findings aren't definitive, and there's still hope for the medications known as ivacaftor (brand name Kalydeco) and lumacaftor, according to ...

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Cystic Fibrosis

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