URGENT NEED FOR ACTION:Help to expedite FDA review of Tezacaftor/Ivacaftor(VX-661)

jricci

Super Moderator
Last week, CFRI’s executive director, Sue Landgraf, met with Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research at the United States Food and Drug Administration (FDA) in Washington DC, to encourage that the FDA expedite the review and approval of new drugs that can benefit the CF community. They discussed FDA policies and procedures around this topic and the possibility of expediting Vertex’s tezacaftor/ivacaftor combination treatment that met both Phase Three trials' primary endpoints, with statistically significant improvement in lung function and no adverse side effects for those with CF that have two copies of the F508del mutation, as well as those that have one copy of the F508del mutation and one residual CFTR mutation.

Dr. Woodcock encouraged members of our CF community to contact her to share their opinion on this issue. Let’s keep up the momentum from last week’s meeting. Thank you for writing to Dr. Woodcock today to ask that the FDA approve – as quickly as possible – new medications that have successfully met the clinical trial endpoints. Lives are depending on them.

Click on this link to have your voice heard:

https://secure3.convio.net/cfri/site/Advocacy?cmd=display&page=UserAction&id=205
 

jricci

Super Moderator
Sorry for the delay- I missed your response somehow.

Yes, definitely-- pass on to others with CF

I also hope that Vertex would start a specific Expanded Access Program for VX-661 for those with severe disease while waiting for FDA review and approval.
 
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