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Vertex Should Expect Approvals For Its Cystic Fibrosis Medications

Vertex Pharmaceuticals (NASDAQ:VRTX) is a large (market cap of $26.49 billion) global biotechnology company with clinical development programs focused on cystic fibrosis (CF) and other serious and life-threatening diseases. Approximately between 70,000 and 100,000 people worldwide have CF, a rare hereditary disorder. CF affects the epithelial cells that line the surfaces and cavities of body organs, including the male genital tract, intestines, lungs, pancreas and sweat glands. The severity of the disease varies by CF transmembrane conductance regulator (CFTR) mutations and environmental factors; CF often starts with bowel obstruction in 15-20% of CF newborns, and ends in early childhood death as a result of progressive obstructive lung disease with airway stretching and widening caused by mucus blockage. Older survivors typically suffer from increasingly frequent hospitalizations for pulmonary disease and/or a 95% male infertility rate in young adulthood. Fewer than 15% of CF individuals have a functioning pancreas, but those who do live much longer (median of 56 years in 1995). Because the build-up of thick secretions in the pancreas prevents the release of digestive enzymes, people with CF need lifelong pancreatic enzyme supplements. Nonetheless, medicinal advances have helped increase the median lifespan of CF patients in the U.S. from 33.4 years in 2003 to 40.7 years today, but the median age of death remains at 27…