Vertex’s Combo Drug Orkambi Faces FDA Doubts About Lumacaftor’s Benefit
This article was originally published in The Pink Sheet Daily
Executive Summary
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.
You may also be interested in...
Vertex Phase III For CF Combo Shows Power Of “Breakthrough” Designation
Pivotal trials for the combination of Kalydeco and VX-809 will measure safety and efficacy over 24 weeks, whereas Kalydeco monotherapy gained FDA approval on the basis of 56 weeks of data; Vertex and FDA developed the protocol together following a recent “breakthrough therapy” designation for the two-drug combo.
FDA’s First Breakthrough Designations Continue Vertex Kalydeco’s Regulatory Successes
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.
BrainStorm’s Phase III Data Do Not Support Clinical Benefit Of NurOwn In ALS, US FDA Says
Agency’s unusual public statement about an investigational product's development status may be aimed at countering bullish comments from the sponsor; FDA cites failure of pivotal trial on the primary and secondary endpoints and ‘modest excess in deaths’ with the cell therapy.
Need a specific report? 1000+ reports available
Buy Reports
Register for our free email digests: