02/01/2012
FDA Approval of Kalydeco!

We have just learned the Food and Drug Administration announced its approval of Kalydeco(tm) (previously known as VX-770) for CF patients with the G551D mutation.

 As the first drug to address the defective CFTR protein, Kalydeco represents a major treatment advance for the cystic fibrosis community. Patients with the G551D mutation who took Kalydeco in clinical trials showed:

 -          A significant improvement in lung function;

 -          A significant reduction in sweat chloride to below the diagnostic level for CF;

 -          Fewer pulmonary exacerbations;

 -          Patient-reported reduction in respiratory symptoms (wheezing, cough, congestion);

 -          Improved weight gain; and

 -          No safety issues.

 We applaud the FDA's swift review of Kalydeco, which underscores the agency's confidence in the safety of the drug, the robustness of the clinical results, and the drug's importance to the health and quality of life for eligible patients.

 For more information, please read our web story, which contains links to the Foundation, Vertex and FDA press releases: http://www.cff.org/aboutCFFoundation/NewsEvents/2012NewsArchive/1-31-FDA-Approves-Kalydeco.cfm

 To help you answer questions from your patients and families about today's news, we have also developed FAQs: http://www.cff.org/treatments/Therapies/Kalydeco/.  People with CF and their families who have additional questions about the Kalydeco news should call 1-800-FIGHT-CF or write to info@cff.org>, and a member of our clinical team will be happy to assist them.

 This achievement would not be possible without your unwavering commitment to people with CF and the families you serve.

 Thank you for giving your time and talent to this effort, and for all you do every day to help patients live longer, healthier lives.

 Thank you again,

 Preston W. Campbell, III, M.D.

Executive Vice President for Medical Affairs