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KALYDECO™ CF benefit ‘persists beyond treatment’


Benefit from the new cystic fibrosis (CF) treatment KALYDECO™ (ivacaftor) last for two times as long as the treatment is administered, a study suggests. 

A long-term follow-up study has shown that  improvements in lung function (forced expiratory volume in one second, FEV1), respiratory symptoms and weight gain among people treated with KALYDECO™ (ivacaftor) for 48 weeks in one of two pivotal studies (STRIVE or ENVISION) were durable for up to 96 total weeks.

The ongoing PERSIST extension study enrolled people with cystic fibrosis (CF) ages six and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and who completed treatment in the Phase 3 STRIVE or ENVISION studies (KALYDECO and placebo treatment groups) and met certain other eligibility criteria. 

A total of nine presentations on KALYDECO were presented at the 35th European Cystic Fibrosis Society (ECFS) Conference in Dublin, June 6 to 9, 2012. 

“KALYDECO has fundamentally changed the way we approach the development of new medicines for cystic fibrosis by targeting the underlying cause of the disease,” said Chris Wright, M.D., Ph.D., Senior Vice President of Global Medicines Development and Affairs at Vertex. 

“We are working hard to understand which additional patients may be helped by KALYDECO and simultaneously explore potential new medicines that may be able to help many more people with CF.” 

KALYDECO is the first medicine to treat the underlying cause of CF, a rare, genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. 

In people with the G551D mutation, KALYDECO helps the defective CFTR protein function more normally. 

An estimated 1200 people in the United States and 1100 people in Europe with CF have at least one copy of the G551D mutation.

KALYDECO was approved by the U.S. Food and Drug Administration (FDA) in January 2012 for use in people with CF ages six and older who have at least one copy of the G551D mutation in the CFTR gene. 

Vertex recently received a positive opinion from the European Committee for Medicinal Products for Human Use (CHMP) recommending the approval of KALYDECO.

Vertex Pharmaceuticals



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