Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 clinical trial data for Trikafta® (elexacaftor/tezacaftor/ivacaftor) in children 6-11 years old with cystic fibrosis.
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The Cystic Fibrosis Foundation has been carefully following the story of a 10-year-old girl with cystic fibrosis who has severe lung disease and has been on a pediatric lung transplant list for 18 months. Her family has organized an online petition and filed a lawsuit to help her receive an adult donor lung.
Today, Vertex Pharmaceuticals Inc. announced positive Phase 3 study results for Trikafta® (elexacaftor/tezacaftor/ivacaftor) for people with cystic fibrosis ages 12 years and older who have one copy of the F508del mutation and one gating or residual function mutation.
The U.S. Food and Drug Administration has approved the use of Kalydeco® (ivacaftor) for infants with cystic fibrosis as young as four months who have certain mutations.
The U.S. Food and Drug Administration (FDA) has approved TOBI® Podhaler™ (tobramycin inhalation powder), a dry powder formulation of the antibiotic TOBI, to treat lung infections caused by the bacteria Pseudomonas aeruginosa (P. aeruginosa).
The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.
The U.S. Food and Drug Administration (FDA) announced that it has approved Ultresa™ delayed-release capsules, a pancreatic enzyme replacement therapy. Ultresa (pancrelipase) is manufactured by Aptalis Pharma.