Cystic Fibrosis: Clinical Assets In Development
The early-stage CF pipeline is full of opportunity for many companies.
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Intellectual property licensed from UCSF focuses on non-mutated, wild-type CFTR activators and inhibitors, thought to offer potential in ophthalmic, renal and gastrointestinal indications.
Vertex is spending $160m to acquire Concert's "enhanced" version of its own blockbuster cystic fibrosis treatment Kalydeco, known as CTP-656, in a defensive move that could allow the company to simplify its dosing regimens and differentiate its products against future competition.
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.