Vertex Receives European CHMP Positive Opinion for ORKAMBI® (lumacaftor/ivacaftor) for Treatment of Children with Cystic Fibrosis Aged 2 to 5 Years Old with Most Common Form of the Disease

Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ORKAMBI^® (lumacaftor/ivacaftor) for the treatment of people with cystic fibrosis (CF) aged 2 to 5 years old who have two copies of the F508del mutation, the most common form of the disease.

If the European Commission issues a favorable adoption of the EMA CHMP opinion for the extension of indication, lumacaftor/ivacaftor will be the first and only medicine approved in Europe to treat the underlying cause of CF for patients aged 2 to 5 years old who have two copies of the F508del mutation.

“Cystic fibrosis is a chronic, progressive disease and it is important to treat early to ensure the best possible outcomes for patients,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Today’s announcement brings us one step closer to providing more young children with a treatment that addresses the underlying cause of their disease. It also marks another important milestone on our path towards our goal of treating every person with this rare and life-limiting disease.”

The submission was supported by data from a Phase 3 open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile in these pediatric patients generally consistent with that in patients aged 6 years and older.

Lumacaftor/ivacaftor is already approved in Europe for the treatment of CF in patients aged 6 and older who have two copies of the F508del mutation.

About CF
Cystic fibrosis is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene. Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the mid-to-late 20s.

About the Phase 3 open-label safety study
This CHMP positive opinion is based on a Phase 3 open-label safety study in 60 patients that showed treatment with lumacaftor/ivacaftor was generally well tolerated for 24 weeks, with a safety profile similar to that in patients aged 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24 (mean decrease in sweat chloride from baseline of 31.7 mmol/L; 95% CI: -35.7, -27.6, n=49). Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study. The most common adverse event (≥30% overall) was cough (63%); most adverse events were mild or moderate in severity. Four patients experienced serious adverse events (2 infective pulmonary exacerbations of cystic fibrosis, 1 gastroenteritis viral, 1 constipation) and three patients discontinued treatment due to elevated transaminases without concurrent elevations in total bilirubin. These findings were presented at the 41^st European Cystic Fibrosis Society Conference in June 2018.

About ORKAMBI ^® (lumacaftor/ivacaftor)
Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.

About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.

Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for nine years in a row.

Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kewalramani’s statement in the third paragraph of this press release. While Vertex believes the forward-looking statements contained in this press release are accurate, there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that Vertex could experience unforeseen delays in obtaining the European Commission favorable adoption of the EMA CHMP opinion for the extension of indication and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

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