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Kalydeco News

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 and older with cystic fibrosis and a [specific gene mutation linked to the disease]. This novel therapy could substantially impact these children's lives, potentially opening the way to even greater progress in years to come." Cystic fibrosis is a life-threatening genetic disease that destroys the lungs and digestive system. More than 70,000 people worldwide have cystic fibrosis, the researchers said. One expert explained that ivacaftor has been considered a big advance in the treatment of the ... Read more

Related support groups: Cystic Fibrosis, Orkambi, Kalydeco, Ivacaftor/lumacaftor, Ivacaftor, Cystic Fibrosis with Ileus

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 of the San Francisco-based Gladstone Institutes, a biomedical research organization. "As shown by our analysis, new treatments depend upon a broad base of scientific knowledge plus special contributions from a few exceptional scientists," Williams said in an institute news release. For anyone suffering from an illness, the dream word is "cure." True cures for disease remain rare, though. But, in the new study the Gladstone team traced the long investigative paths linking generations of ... Read more

Related support groups: Diabetes, Type 2, Cancer, Heart Failure, Parkinson's Disease, Congestive Heart Failure, Diabetes, Type 1, Alzheimer's Disease, Diabetes Mellitus, Yervoy, Diagnosis and Investigation, Parkinsonism, Orkambi, Ipilimumab, Ivacaftor, Kalydeco, Ivacaftor/lumacaftor

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 release. Having two copies of this gene mutation is the leading cause of cystic fibrosis, accounting for about half of all cases. "The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis," Dr. John Jenkins, director of the Office of New Drugs, Center for Drug Evaluation and Research, said in the news release. "Today's approval significantly broadens the availability of targeted treatments for the specific defects that cause cystic ... Read more

Related support groups: Cystic Fibrosis, Orkambi, Kalydeco, Ivacaftor/lumacaftor, Ivacaftor

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 treating certain people with cystic fibrosis, according to the researchers. "Just a few years ago, ivacaftor became the only FDA-approved drug for the genetic defect in cystic fibrosis, but it only works for genetic mutations found in a small portion of cystic fibrosis patients. Our study showed that combining ivacaftor with lumacaftor helps patients with the most common cystic fibrosis mutation. This is an exciting step forward," said study co-author Dr. Susanna McColley in a news release from ... Read more

Related support groups: Cystic Fibrosis, Kalydeco, Ivacaftor

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

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, S549R and R117H). Prior to today's approval, Kalydeco was approved in the United States for people ages 6 and older with these mutations. There are approximately 300 children in the United States ages 2 to 5 who have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations that result in a gating defect in the CFTR protein. A new weight-based oral granule formulation of Kalydeco (50 mg and 75 mg) that can be mixed in soft foods or liquids ... Read more

Related support groups: Cystic Fibrosis, Kalydeco, Ivacaftor

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) gene. Today's approval follows a recommendation by the FDA's Pulmonary-Allergy Drugs Advisory Committee (PADAC) to approve the medicine in this group of people with CF. Kalydeco is now approved for use in the U.S. in people ages 6 and older with CF with one of the following ten mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The approval is based on previously announced data from a Phase 3 study of ivacaftor that enrolled 69 people with CF ages 6 and older ... Read more

Related support groups: Cystic Fibrosis, Kalydeco, Ivacaftor

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 the study's senior author. "The development of drugs like ivacaftor and lumacaftor is undoubtedly a step forward, but our study suggests that more work will need to be done before we can realize the full potential of these drugs," said Martina Gentzsch, an assistant professor with the department of cell biology and physiology at the University of North Carolina, Chapel Hill. "Fortunately, we now have a better understanding of some of the potential pitfalls to these drug combinations and a means to ... Read more

Related support groups: Cystic Fibrosis, Kalydeco, Ivacaftor

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