VOTUBIA (everolimus), mTOR inhibitor
RARE DISEASE - New indication
Opinions on drugs - Posted on Mar 23 2015
Reason for request
Substantial improvement when a subependymal giant cell astrocytoma associated with tuberous sclerosis complex is not immediately eligible for surgical resection
VOTUBIA, available as a tablet and dispersible tablet, now has Marketing Authorisation in children under age 3 years in subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex, in patients who require therapeutic intervention but are not eligible for SEGA surgical resection.
This treatment is superior to placebo for reducing the SEGA volume, but has not shown any improvement in the symptoms related to the disease.
Its therapeutic benefit is substantial only to prepare patients for surgery in certain advanced SEGA cases and for patients for whom its severity justifies a surgical procedure, but who are not amenadable to surgery.
Clinical Added Value
|no clinical added value|
Évaluation des médicaments