Pink Sheet is part of Pharma Intelligence UK Limited

This site is operated by Pharma Intelligence UK Limited, a company registered in England and Wales with company number 13787459 whose registered office is 5 Howick Place, London SW1P 1WG. The Pharma Intelligence group is owned by Caerus Topco S.à r.l. and all copyright resides with the group.

This copy is for your personal, non-commercial use. For high-quality copies or electronic reprints for distribution to colleagues or customers, please call +44 (0) 20 3377 3183

Printed By


FDA’s First Breakthrough Designations Continue Vertex Kalydeco’s Regulatory Successes

This article was originally published in The Pink Sheet Daily

Executive Summary

Label expansion efforts for Vertex’s cystic fibrosis therapy will be claims taken through FDA’s new review pathway; the initial product could reach more patients more quickly and serve as a guide to how the agency will handle drugs earmarked for streamlined development and approval.

You may also be interested in...

Vertex’s Orkambi Efficacy Data May Make FDA More Cautious With Other ‘Breakthroughs’

Agency’s trial design recommendations made it difficult to assess lumacaftor’s contribution to the lumacaftor/ivacaftor fixed-dose combination’s efficacy in cystic fibrosis. The experience may color FDA’s future approach to combination product trial designs and how it advises other ‘breakthrough therapy’ sponsors on the evidence likely needed to support approval.

Vertex’s Combo Drug Orkambi Faces FDA Doubts About Lumacaftor’s Benefit

Advisory committee to consider whether Vertex should conduct new clinical trial comparing ‘breakthrough therapy’-designated, fixed-dose combo of lumacaftor and ivacaftor to ivacaftor alone in subgroup of cystic fibrosis patients.

Return Of Vertex’s Ivacaftor To FDA Panel May Signal Efficacy Concerns

Kalydeco raced to market for cystic fibrosis, but effort to get ‘breakthrough’ approval with lumacaftor for much larger patient population may have FDA worrying about the meaningfulness of pivotal efficacy results.

Related Content


Related Companies

Latest Headlines
See All



Ask The Analyst

Ask the Analyst is free for subscribers.  Submit your question and one of our analysts will be in touch.

Your question has been successfully sent to the email address below and we will get back as soon as possible. my@email.address.

All fields are required.

Please make sure all fields are completed.

Please make sure you have filled out all fields

Please make sure you have filled out all fields

Please enter a valid e-mail address

Please enter a valid Phone Number

Ask your question to our analysts