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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

Why Do Some Kids Escape Terrible Genetic Disorders?

Posted 11 Apr 2016 by Drugs.com

MONDAY, April 11, 2016 – Some severe genetic birth defects, like cystic fibrosis, are considered inescapable, automatically dooming children to disease or disability if they inherit a mutated gene from their parents. But researchers now have found rare instances where children have beaten the odds and defied their genetic destiny. Analysis of nearly 600,000 people's genetic makeup revealed 13 healthy individuals who should have suffered a terrible childhood disease due to their genetics, but for some reason did not. Figuring out how certain people are able to dodge their genetics could provide solutions to some of mankind's worst birth defects, said senior researcher Dr. Stephen Friend. He is the president of Sage Bionetworks, a nonprofit biomedical research organization in Seattle. "If you want to develop therapies for prevention, if you want to come up with ways of not just finding ... Read more

Related support groups: Cystic Fibrosis, Diagnosis and Investigation

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

Scientists, Ethicists Debate Future of Gene Editing

Posted 3 Dec 2015 by Drugs.com

WEDNESDAY, Dec. 2, 2015 – What if faulty genes in your DNA could be easily corrected, avoiding the ravages of diseases like cystic fibrosis or certain cancers? That is the promise of gene editing, a new technique being heralded as an enormous advancement in genetic engineering. Scientists say its speed, efficiency and cost-effectiveness make it an excellent tool for replacing rogue genes that cause human suffering and early death. But worries about the technology's darker side have mounted ever since Chinese researchers in April reported results of an experiment to edit a defective gene in a human embryo. For ethical reasons, researchers say they used an abnormal embryo. The team concluded that gene editing isn't yet sufficiently reliable and precise enough to apply the technique in humans. Nevertheless, some scientists and bioethicists worry that gene editing may be used one day to ... Read more

Related support groups: Cancer, Cystic Fibrosis, Diagnosis and Investigation

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, Ivacaftor/lumacaftor, Ivacaftor, Kalydeco

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

Gene Therapy for Cystic Fibrosis Shows Some Promise in Study

Posted 2 Jul 2015 by Drugs.com

THURSDAY, July 2, 2015 – Gene therapy for cystic fibrosis has for the first time shown slight but significant benefit on lung function, new British research reveals. In a randomized trial, patients inhaled molecules of DNA that aimed to replace the defective gene responsible for cystic fibrosis with a healthy, working copy of the gene in the lungs. "Patients who received the gene therapy showed a significant, if modest, benefit in tests of lung function compared with the placebo group, and there were no safety concerns," study senior author Eric Alton of the National Heart and Lung Institute at Imperial College London, said in a news release fromThe Lancet Respiratory Medicine, which published the study. Even though the effect was "inconsistent, with some patients responding better than others, the results are encouraging," Alton said. Cystic fibrosis is a rare, inherited respiratory ... Read more

Related support groups: Cystic Fibrosis, Cystic Fibrosis with Ileus

Cystic Fibrosis Might Be Deadlier for Hispanics, Study Suggests

Posted 30 Jun 2015 by Drugs.com

TUESDAY, June 30, 2015 – Cystic fibrosis is deadlier for Hispanic patients than others, and genetic differences may make Hispanics less likely to benefit from new treatments for the disease, researchers report. "We need to ask if the care model for patients with [cystic fibrosis] is working for this minority group," study author Dr. MyMy Buu, an instructor in pediatric pulmonary medicine at Stanford University School of Medicine in Palo Alto, Calif., said in a university news release. "We want to make sure that what we are doing is not inadvertently causing disparities." The findings reflect an urgent need to identify the factors that contribute to this health disparity, the researchers noted. "This will be crucial to develop treatment regimens that guarantee that all children with [cystic fibrosis] can benefit from early diagnosis and the novel treatments being introduced," study ... Read more

Related support groups: Cystic Fibrosis

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, Ivacaftor, Kalydeco

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, Ivacaftor, Kalydeco

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, Ivacaftor, Kalydeco

FDA Approves PulmoFlow's Kitabis Pak Tobramycin & Nebulizer Combination for Cystic Fibrosis

Posted 16 Dec 2014 by Drugs.com

RICHMOND, Va., Dec. 3, 2014 /PRNewswire/ – On Tuesday, the FDA granted final approval of PulmoFlow, Inc.'s New Drug Application for Kitabis Pak – a co-packaging of generic tobramycin inhalation solution with a PARI LC PLUS® Nebulizer. This is the first nebulized drug and device combination to be approved for patients with cystic fibrosis. Kitabis Pak is in stock and available now through PARI Respiratory Equipment, Inc. Kitabis Pak sets a new standard for nebulized drugs similar to asthma & COPD inhalers where the drug and device are prescribed and dispensed together. The price of Kitabis Pak will be similar to the price of generic tobramycin drug alone. "Patient access to the only approved and proven delivery system for nebulized tobramycin is very important both for the patient and healthcare professional. Knowing that we don't have to worry about the device any longer is a huge ... Read more

Related support groups: Cystic Fibrosis, Tobramycin

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, Ivacaftor, Kalydeco

FDA Medwatch Alert: Acetylcysteine Solution 10 Percent: Recall - Glass Particles in Product

Posted 17 Feb 2014 by Drugs.com

ISSUE: Ben Venue Laboratories notified health professionals and their medical care organizations of a nationwide product recall of Acetylcysteine Solution 10%, manufactured for Roxane Laboratories, NDC #0054-3025-02 – Lot 2005479 – Exp. Date March 2014. The recall was initiated on February 14, 2014 after the discovery of a single visible glass particle in a vial within the lot listed above. The nationwide voluntary recall is to the patient level. This recall is limited to lot number 2005479. Glass particles can cause airway obstruction resulting in symptoms of choking, wheezing, difficulty breathing, coughing and potentially hemoptysis. Use of an inhaled product with glass particles has the potential to cause choking which could be life-threatening. Aerosolization of small glass particles in the airways could result in recurrent infections (due to obstruction of airways, and decreased cl ... Read more

Related support groups: Cystic Fibrosis, Acetylcysteine, Mucomyst, NAC, Mucomyst-20, Mucomyst-10, Parvolex, Solgar NAC, Acys-5, Acetadote

FDA Allows Marketing of Four “Next Generation” Gene Sequencing Devices

Posted 19 Nov 2013 by Drugs.com

November 19, 2013 – Today the U.S. Food and Drug Administration allowed marketing of four diagnostic devices that can be used for high throughput gene sequencing, often referred to as “next generation sequencing” (NGS). These instruments, reagents, and test systems allow labs to sequence a patient’s DNA (deoxyribonucleic acid). The new technology also gives physicians the ability to take a broader look at their patients’ genetic makeup and can help in diagnosing disease or identifying the cause of symptoms. “NGS is changing the way we look at genomics,” said Alberto Gutierrez, Ph.D., director of the Office of In Vitro Diagnostics and Radiological Health in FDA’s Center for Devices and Radiological Health. “Before NGS, sequencing genes associated with a particular disease was a long and costly process. Today, we have the capability to read and interpret large segments of DNA very quick ... Read more

Related support groups: Cystic Fibrosis

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