Kalydeco Label Expansion Could Set New Precedent On FDA Flexibility
Agency must decide whether to approve for cystic fibrosis patients with R117H mutation even though the pivotal trial failed and age-related subgroup analyses produced conflicting results. Vertex picked up an advisory committee endorsement despite panelists’ concerns about suboptimal results in pediatric patients.
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Kalydeco, an oral CFTR potentiator, becomes the first approved drug to address the underlying cause of cystic fibrosis, rather than just treating the disease’s symptoms.
AMAG outlines plans for retrospective real-world evidence studies, and possibly a new placebo-controlled trial with a primary endpoint of preterm birth <32 weeks, in a filing opposing the Center for Drug Evaluation and Research’s proposal to withdraw the drug.
Office of New Drugs Director Peter Stein tells the Pink Sheet that last phase of massive reorg was complicated by pandemic restrictions that forced review staff to work remotely, but the structural overhaul resulted in smaller, more nimble review groups and the breaking down of silos that ultimately helped the agency’s response to the public health crisis.