“For the first time in over a decade, a striking silence fills the Cheevers' barn-style home,” begins a story in the September issue of the science magazine Discover.
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The U.S. Food and Drug Administration (FDA) announced today that it has approved the use of ivacaftor (Kalydeco™) to treat people with cystic fibrosis ages 6 and older who have the R117H mutation.
The CF Foundation has successfully completed a sale of its remaining stake in royalties related to Vertex's CFTR modulators for an upfront payment of $575 million and a potential future payment of $75 million, bringing additional resources to the fight against CF.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
On Aug. 27, leaders from the Cystic Fibrosis Foundation, along with several people from the CF community, participated in a virtual public meeting hosted by the Institute for Clinical and Economic Review to provide insights and expertise on the value of CFTR modulators.
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The European Medicines Agency (EMA) announced today that it has recommended the approval of the CF drug Kalydeco™ for people with the G551D mutation ages 6 and older in the European Union.