Vertex Pharmaceuticals' ivacaftor reduced pulmonary flare-ups by 55% compared with a placebo. For now it applies to those with a certain genetic mutation, but the pool of patients could grow.

Los Angeles Times, By Amina Khan, November 2

All her life, Lindsay Shipp knew that she was dying. As a baby, she would cry after eating, and salt collected on her forehead. The diagnosis was cystic fibrosis, an incurable genetic disease that, at the time, meant a life expectancy of 18 years.

The disease, which affects 30,000 people in the United States, hinders the movement of salt in the body. Because of this, the pancreas fails soon after birth, patients cannot properly digest food, and their airways fill with mucus, leaving them vulnerable to lung infections and other problems. The current average life expectancy is now 37.

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But since she started taking an experimental drug as part of a closely watched clinical trial, her outlook has changed. The drug, called ivacaftor, cleared her lungs and allowed her to add a healthy 15 pounds to her 5-foot, 100-pound frame. On the day in February of last year when a regular checkup revealed her lungs were functioning at 96% — bringing her to essentially the same level as a healthy person — Shipp collapsed on a bench outside the hospital and sobbed with relief. "I realized I would have a full life at that moment," she said.

The trial results, published in Thursday's edition of the New England Journal of Medicine, showed that ivacaftor reduced the incidence of pulmonary exacerbations — sudden, serious flare-ups that can send patients to the hospital and cause irreversible lung damage — by 55% compared with a placebo. The study of 161 patients also found that patients who took the drug saw their lung function improve 10.4% and gained nearly 6 pounds over 48 weeks.