Kalydeco, For Cystic Fibrosis, Wins First Annual Forbes Breakthrough Drug Award

Kalydeco, a treatment for cystic fibrosis developed by Vertex Pharmaceuticals VRTX +0.81% with support from the Cystic Fibrosis Foundation , was awarded the first Forbes Breakthrough Drug Award today.

For dramatically improving the lives of patients and conquering a scientific challenge that had vexed researchers for decades.

The award was presented to Jeffrey Leiden, Vertex’s chief executive, and Fred Van Goor, a scientist who played a key role in inventing Kalydeco at the Forbes Healthcare Summit in New York.

“This drug is the first effective treatment for a deadly disorder (cystic fibrosis),” says Steven Nissen of the Cleveland Clinic, one of an expert panel convened to pick an honoree. “Although I do not care for these children, I am well aware of the devastating effects of this disease on patients and their families.”

Helping me choose, aside from Nissen: Peter Bach, of Memorial Sloan-Kettering, who has criticized the pharmaceutical industry’s pricing practices; Richard Evans, of SSR Research, a long-time industry analyst; Bernard Munos, a consultant who studies why it is so difficult to invent new medicines. I voted, too.

The panel unanimously chose to look at a period of several years, because not every year brings a breakthrough and some bring two. And we chose not to consider medicines until they had been on the market for a few years, because sometimes new drugs turn out to be either less or more important than they seem. Each panelist made his own choices, which we then discussed. We ranked the contenders based on that voting.

Kalydeco was the clear winner, appearing high on each judge’s list and getting a total of four stars on our rating scale.. But there were other important medicines approved during this time period, too. The runners up, in descending order

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