Vertex Pharmaceuticals Makes Breakthrough on Cystic Fibrosis Drug

NEC member Vertex Pharmaceuticals has created a triple drug regime that has proven to be an effective treatment for cystic fibrosis patients who are genetically resistant to all other drugs on the market.

Vertex recently received the data from the second phase of testing three separate triple combinations of cystic fibrosis drugs and they were overwhelmingly positive. There was a highly statistically significant 10 percentage point improvement, adjusted for placebo, in patients who are the hardest to treat because of a genetic mutation.  Of the approximately 75,000 cystic fibrosis patients around the world, it is estimated that 24,000 carry the mutated gene that Vertex has developed a treatment for.  Vertex is planning for one or two late stage clinical trials in early 2018.

“Everything we saw in the lab played out in the clinic,” Vertex’s Chief Executive Officer Jeffery Leiden said. “It’s one of those very rare moments in biotech. This is the most significant advance to date in our ability to fundamentally change the course of the disease for up to 90 percent of all patients. We have an incredible sense of urgency now.”

The New England Council congratulates Vertex on this breakthrough and looks forward to the results of the Phase 3 studies.  Read more in The Boston Globe and on Vertex’s website.

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