Pink Sheet is part of Informa PLC

This site is operated by a business or businesses owned by Informa PLC and all copyright resides with them. Informa PLC’s registered office is 5 Howick Place, London SW1P 1WG. Registered in England and Wales. Number 8860726.

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


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

Related Content





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