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FDA grants approval to ivacaftor for treatment of cystic fibrosis in infants
USA: The US Food and Drug Administration (FDA) has given approval to ivacaftor for treatment of cystic fibrosis in infants aged four months to less than six months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator (CFTR) gene. Kalydeco (ivacaftor) is already approved by the FDA for treating cystic fibrosis in patients aged 6 months and older.
"Initiating therapy that treats the underlying cause of cystic fibrosis as early as four months of age may have the potential to modify the course of the disease," said Margaret Rosenfeld, M.D., MPH, Seattle Children's Research Institute and Department of Pediatrics, University of Washington School of Medicine.
This FDA approval is based on the results from 24-week Phase 3 open-label safety cohort (ARRIVAL) trial consisting of 6 children with CF ages four months to less than six months who have one of 10 mutations in the CFTR gene (G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or R117H). This cohort demonstrated a safety profile similar to that observed in older children and adults.
"Since the initial approval of KALYDECO more than eight years ago, we have continued to advance our clinical development program with the goal of treating the underlying cause of cystic fibrosis as early in life as possible," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "Today's approval is a testament to our relentless efforts, alongside the clinical and scientific community, to reach all people with CF who may benefit from our medicines."
About Cystic Fibrosis
Cystic fibrosis, a rare, progressive, life-threatening disease, results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. It leads to severe respiratory and digestive problems as well as other complications such as infections and diabetes. Cystic fibrosis is caused by a defective protein that results from mutations in the CFTR gene. While there are approximately 2,000 known mutations of the CFTR gene, the most common mutation is the F508del mutation.
MSc. Biotechnology
Medha Baranwal joined Medical Dialogues as an Editor in 2018 for Speciality Medical Dialogues. She covers several medical specialties including Cardiac Sciences, Dentistry, Diabetes and Endo, Diagnostics, ENT, Gastroenterology, Neurosciences, and Radiology. She has completed her Bachelors in Biomedical Sciences from DU and then pursued Masters in Biotechnology from Amity University. She has a working experience of 5 years in the field of medical research writing, scientific writing, content writing, and content management. She can be contacted at  editorial@medicaldialogues.in. Contact no. 011-43720751
Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751