On January 31st the Cystic Fibrosis Foundation announced that the life-saving drug Kalydeco has been approved by the FDA.
Nashville CF patients will now have access to the drug that is considered a breakthrough drug for Cystic Fibrosis. Kalydeco, or VX-770, was submitted to the FDA in October after years of trials and put on the fast track for approval due to it’s immediate need.
Kalydeco works to correct a specific CF mutation, G551D, which affects about 4% of those with CF.
Researchers are hopeful that work with VX-770 will lead to the creation of further treatments to help those who won’t qualify for this particular drug. Work is already under way for others.
The research is backed by the Cystic Fibrosis Foundation, one of the top rated charities in the world for it’s excellent financial management and the many advancements they’ve made in quality of life for those with the disease. The Nashville CFF chapter holds fundraisers throughout the year, including it’s popular Great Strides fundraising walk held each year. This year’s Great Strides walk in Nashville is scheduled for May 20, 2012. Go to http://www.cff.org/Great_Strides/ to donate or register.
From October 2011
VX-770 which has been in trial for several years now, has shown from the beginning to be highly effective in treating certain forms of Cystic Fibrosis. VX-770 which was submitted October 19 for approval to the FDA under the drug company Vertex Pharmaceuticals, will be introduced to the public as Kalydeco.
While the treatments currently available to CF patients treat only the symptoms of CF, Kalydeco treats the underlying cause, preventing symptoms from the offset and decreasing the necessity of more interventions.
The Cystic Fibrosis Foundation has worked in conjunction with Vertex Pharmaceuticals to pinpoint the genes that cause CF and to develop VX-770.
Kalydeco is slotted to be approved sometime in 2012 for patients over the age of 6 and who carry at least one copy of the G551D mutation of Cystic Fibrosis. Vertex has requested priority review from the FDA, which would shorten the wait time before it's available to the public.
For those cases that are more medically urgent, it may be possible to receive treatment now under a special expanded access program. For more information contact a CF Foundation-accredited care center.
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