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FDA Approves Orkambi (lumacaftor/ivacaftor) for Use in Children with Cystic Fibrosis Ages 6 through 11 who have Two Copies of the F508del Mutation

Posted 29 Sep 2016 by Drugs.com

BOSTON--(BUSINESS WIRE) September 28, 2016 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) approved Orkambi (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. People with this mutation represent the largest population of those with CF, a rare, life-threatening disease. Orkambi is the first and only medicine to treat the underlying cause of CF for people with this mutation. It was previously approved by the FDA for use in people ages 12 and older with two copies of the F508del mutation. With today's approval, approximately 11,000 people with CF are eligible for treatment with Orkambi in the United States. Orkambi will be available for eligible children ages 6 through 11 in the United States as soon as possible. Vertex also today lowered ... Read more

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

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, Ivacaftor, Cystic Fibrosis with Ileus, Kalydeco, Ivacaftor/lumacaftor

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, Kalydeco, Ivacaftor/lumacaftor, Ivacaftor

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

FDA Approves Orkambi (lumacaftor/ivacaftor) for Cystic Fibrosis

Posted 2 Jul 2015 by Drugs.com

July 2, 2015 – The U.S. Food and Drug Administration today approved the first drug for cystic fibrosis directed at treating the cause of the disease in people who have two copies of a specific mutation. Orkambi (lumacaftor 200 mg/ivacaftor 125 mg) is now approved to treat cystic fibrosis (CF) in patients 12 years and older, who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF. “The FDA encourages manufacturers to develop new and innovative treatments for serious rare diseases like cystic fibrosis,” said John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. “Today’s approval significantly broadens the availability of targeted treatments for the specific de ... Read more

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

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