Does Xenazine's Effect On Chorea Outweigh Risks? Ask The Patients
This article was originally published in Pharmaceutical Approvals Monthly
Executive Summary
Including patient-reported outcomes in the pivotal Xenazine trials could have provided valuable insight into the "net benefit" of the drug as FDA struggled to weigh the difficult combination of positive effects on one major symptom of Huntington’s disease and potential negative effects on other symptoms, FDA review documents suggest.
You may also be interested in...
Revisiting Xenazine’s Extraordinary Review: A Study In FDA Flexibility
Unmet need is high in Huntington’s disease and the protracted review of Xenazine – the only drug cleared for the disease by FDA – suggests regulatory precedent for approval based on just one efficacy endpoint and with lots of development and safety baggage.
FDA's First Step In Xenazine Review: Work Around Clinical Evidence Guidance
Shepherding the orphan Huntington’s chorea drug Xenazine through a three-year review, top-level FDA reviewers invoked the concept of "confirmatory evidence" to circumvent the agency’s preference for two randomized clinical trials to support new drug approval.
Advisory Panel Questions Huntington’s Scale During Review Of Xenazine
That Prestwick Pharmaceutical's Xenazine's significant efficacy in treating the involuntary movements (chorea) of Huntington's Disease did not translate to a benefit over placebo for non-motor endpoints may be the fault of an inadequate scale to measure functionality, an FDA panel said