The Price Of Inspiration
Forbes|September 5, 2017

Vertex Pharmaceuticals’ New $200,000-A-Year Cystic Fibrosis Drug Could Help 90% Of Patients Breathe. It’s An Amazing—And Profitable— Innovation. But Will That Windfall Spur The 28-Year-Old Startup To Do It Again?

Matthew Herper
The Price Of Inspiration

Technically, Mark Sleeper doesn’t know for sure he didn’t get the placebo, but nothing in his 27 years of fighting the deadly lung disease cystic fibrosis has made him feel as good as the pill he recently took in a clinical trial. “It was kind of mind-blowing,” he says.

The energy he usually spent coughing and simply breathing was suddenly available for use in daily living. At first, he stayed up for two days. After 15 days on the treatment, his lung capacity, measured by the amount of air he can breathe out in a second, went from 70% of normal for a man his age to normal. “With what I experienced, I want the drug to be approved, because it was pretty radical,” he says. “It was pretty cool to just be on it for 15 days. It’s amazing to think of what it would be like to take it for a month.”

Or a lifetime. Study results released in July for three different but similar drug combinations, all developed by Boston’s Vertex Pharmaceuticals, back up Sleeper’s experience, showing the amount of air patients could exhale in a second increased by ten percentage points or more—an unheard-of amount. There’s only one precedent for results like this in CF: another Vertex drug, Kalydeco, approved for use in 6% of the 30,000 Americans with the disorder. The new combinations could bring a similar benefit to almost all cystic fibrosis patients.

“There’s going to be a paradigm shift in the way we think about treating this disease,” says Jeffrey Leiden, Vertex’s chief executive. “It’s not going to be four different medicines for different subsets of patients, or incremental improvements. It’s going to jump to the end. And it’s going to be Kalydecolike efficacy or better, and 90% of the patients getting a single regimen.”

This story is from the September 5, 2017 edition of Forbes.

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This story is from the September 5, 2017 edition of Forbes.

Start your 7-day Magzter GOLD free trial to access thousands of curated premium stories, and 8,500+ magazines and newspapers.