Cystic Fibrosis: Successes, Opportunities And Challenges
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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A deeper understanding of rare diseases – how they differ across geographies and ethnicities, or which genes and biomarkers are most useful targets for new treatments – can help to accelerate and de-risk clinical trials. On the commercial front, patient data and insights could mean the difference between a category leader and a failure.
Semma closed a $114m Series B round to fund development of insulin-producing cells. Also, Tesaro and Catalyst are among recent public company financings, while Otonomy shifted gears, laying off sales staff.
Among the antibiotics that have garnered a qualified infectious disease product designation, a collection of late-stage products are targeted at local delivery, especially inhaled and vaginal formulations.