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NHS In England Funds KALYDECO™ (ivacaftor), The First Medicine To Treat The Underlying Cause Of Cystic Fibrosis, For People With A Specific Genetic Mutation (G551D)

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that a decision has been made by the National Health Service (NHS) in England to fund KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), for people ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene. Cystic fibrosis is a rare genetic disease for which there is no cure. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the G551D mutation, ivacaftor helps the defective CFTR protein function more normally. In England, several hundred of the nearly 8,000 people with CF are believed to have at least one copy of the G551D mutation.

Today’s announcement concludes a comprehensive and robust clinical and economic evaluation of the medicine by the NHS in England. Vertex has agreed to a patient access scheme with the NHS, the details of which remain confidential. Vertex will make ivacaftor available to eligible people with CF as quickly as possible and anticipates reimbursement to begin in the second quarter of 2013.

“We are pleased to have been able to work with the NHS to receive a decision to fund ivacaftor so quickly,” said Simon Bedson, General Manager of Vertex Europe. “We will be working with the NHS to help them to implement this decision as quickly as possible to ensure that people with cystic fibrosis who are eligible for ivacaftor can access it without delay.”

“Ivacaftor changes the way we treat cystic fibrosis because now, for the first time, we are able to target the underlying cause of the disease in those with the G551D mutation, instead of just the symptoms and complications,” said Jane Davies, M.D., Royal Brompton Hospital and Imperial College, London.

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