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Cystic Fibrosis: Successes, Opportunities And Challenges

 
• By Lucie Ellis-Taitt

What is next for the treatment of cystic fibrosis, a rare disease market that has boomed in recent years on the back of new drug approvals with hefty price tags? Pipelines are full, but securing reimbursement is a challenge.

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Vertex Pharmaceuticals Inc.has quickly established itself as the one to beat across the cystic fibrosis market, and the company is expecting critical data readouts for some of its pipeline products this year. Still, the early-stage pipeline for this rare disease indication is plentiful, including activity from Vertex's former hepatitis C rival, AbbVie Inc.

In parallel to new therapies coming to the fore only a few years after the launch of Vertex's standard-of-care-altering Kalydeco...

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