Expert Interview
A Second Look: Discussing Denali Therapeutics’ investigational brain-penetrant enzyme therapy, tividenofusp alfa, for mucopolysaccharidosis type II (Hunter syndrome)
Ticker(s): DNLI, TAKPediatric geneticist, metabolic disease specialist, or pediatric neurologist with experience managing patients with Hunter syndrome / MPS II and familiarity with enzyme replacement therapy and CNS disease progression.
How are you currently managing patients with Hunter syndrome, and what limitations do you see with existing therapies in addressing neurologic versus peripheral disease manifestations?
What is your interpretation of the clinical data for tividenofusp alfa, particularly the reported effects on CNS biomarkers, peripheral biomarkers, and clinical measures such as cognition, adaptive behavior, hearing, or organ involvement?
Added By: sara_adminHow clinically meaningful do you believe a brain-penetrant enzyme replacement therapy could be for patients with MPS II, especially if initiated before advanced neurologic impairment?
Added By: sara_adminHow do you expect to discuss the option of switching with your patients? Do you expect to have these discussions with stable patients? Right away? At annual check-in?
Added By: sara_adminDo you have any concerns about antibody development/Neutralizing antibodies that could impact the efficacy of Avlayah? How will that be assessed?
Added By: sara_adminWhat is on your "checklist" for evaluating patients for whether they should switch to Avlayah
Added By: sara_adminwill you proactively call in patients for an appointment to switch them or wait for their annual visit - especially if they don't reach out to you immediately
Added By: sara_adminDo you expect any of your patients remaining on Elaprase? Which patients? Why?
Added By: sara_adminDo geneticists at small community hospitals have familiarity around new therapies? Do they treat Hunter patients or refer them out to COEs?
Added By: sara_adminHow concerning are the precautions around membranous nephropathy and anemia listed on the label?
Added By: sara_adminWhat additional data do you wish/hope to have to support your decision to prescribe Avlayah?
Added By: sara_adminCan a patient return to Elaprase if they can not tolerate Avlayah? Will antibodies make that impossible?
Added By: sara_adminAre IRRs a concern with Avlayah?
Added By: sara_adminAre You Interested In These Questions?
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