Expert Interview
Discussing the potential of Praxis Precision Medicines' Ulixacaltamide in Essential Tremors following NDA submission
Ticker(s): PRAXInstitution: University of Louisville
- Professor of Neurology, Medical Geneticist, Endowed Chair of Parkinson’s Disease Research, & Director, Movement Disorders Program at University of Louisville.
- Manages 90 patients with Huntington's disease and 35 patients with Friedreich’s Ataxia.
- Research interests are focused on understanding the underlying genetic basis of hereditary spastic paraplegia and essential tremor; clinical interests include surgical therapies for Parkinson's disease, essential tremor and dystonia, and botulinum toxin procedures for dystonia and spasticity.
What are your thoughts on FDA approvability, if IDMC used unblinded data to recommend statistical model changes?
Added By: wilson_adminGiven a majority of discontinued ulixacaltamide patients are TEAE-related, do you think this is an appropriate imputation method the FDA would approve?
Added By: wilson_adminGiven the overall efficacy /safety profile, do you think Ulixacaltamide trial and statistical analysis plan changes pose a risk onapprovability? Do you think the current FDA neurology division would approve Ulixacaltamide?
Added By: wilson_adminHow would FDA approach analyzing Praxis’s ET data set given trial design changes? Is there precedent neurology trials whereendpoints and SAP were modified that lead to an approved drug? (eg. aducanumab) and how does Praxis’ data compare in terms of approvability?
Added By: wilson_adminAre You Interested In These Questions?
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