Iclusig Experience Reflects Regulatory Risk Inherent With Accelerated Approval
This article was originally published in Pharmaceutical Approvals Monthly
FDA reviewers and outside experts discussed at length concerns about cardiovascular and other adverse events in the single-arm PACE trial but believed ponatinib’s impressive efficacy in a difficult-to-treat leukemia population warranted bringing the drug to market. Sales of the tyrosine kinase inhibitor were halted less than 11 months later as safety and dosing concerns continued to pile up.
You may also be interested in...
Hemophilia patient advocate expressed concerns to FDA about ability of hemophilia treatment centers to educate patients about thrombotic microangiopathy and thromboembolic events, unexpected but serious cases of which were seen in the clinical program.
FDA rejected two of the sponsor’s labeling proposals because they failed to adequately address safety issues seen in the PACE trial. Ariad apparently preferred to quantify in labeling only treatment-emergent adverse events that investigators attributed to ponatinib – an approach FDA disdains for single-arm studies.
The period after completion of pivotal trials but ahead of FDA approval provides an opportunity for oncology drug sponsors to conduct badly needed dose comparison trials, a multi-stakeholder panel says at the annual Conference on Clinical Cancer Research.