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Republic Of Ireland Approves Funding For 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 the Health Service Executive (HSE) in the Republic of Ireland will 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.

Today’s decision follows an assessment of the medicine by the National Centre for Pharmacoecomomics (NCPE), which acknowledged the benefits of ivacaftor, including significant improvement in lung function, increased body weight, improvement in quality of life and a 55 percent reduction in pulmonary exacerbations, or periods of worsening respiratory signs and symptoms that often require treatment with antibiotics and hospitalisation. Vertex is working to make ivacaftor available to eligible people in Ireland as quickly as possible.

“We are delighted that the HSE will make ivacaftor available for eligible people with cystic fibrosis in Ireland,” said Simon Bedson, General Manager of Vertex Europe. “We will work with them to help implement this decision to ensure that those who are eligible can access ivacaftor as quickly as possible.”

Ivacaftor was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, Inc., the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation.

About Ivacaftor

Ivacaftor is the first medicine to treat the underlying cause of CF in people with the G551D mutation in the CFTR gene. Known as a CFTR potentiator, ivacaftor is an oral medicine that aims to help the CFTR protein function more normally once it reaches the cell surface, to help hydrate and clear mucus from the airways. Ivacaftor (150mg, q12h) was first approved by the U.S. Food and Drug Administration in January 2012, by the European Medicines Agency in July 2012 and by Health Canada in November 2012 for use in people with CF ages 6 and older who have at least one copy of the G551D mutation in the CFTR gene.

Vertex retains worldwide rights to develop and commercialize ivacaftor. A Marketing Authorization application is under review by the Therapeutic Goods Administration (TGA) of Australia.

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