Primo-indication
Renal angiomyolipoma associated with tuberous sclerosis complex (TSC)Votubia is indicated for the treatment of adult patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC) who are at risk of complications (based on factors such as tumour size or presence of aneurysm, or presence of multiple or bilateral tumours) but who do not require immediate surgery.The evidence is based on analysis of change in sum of angiomyolipoma volume.Subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC)Votubia is indicated for the treatment of patients with subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex (TSC) who require therapeutic intervention but are not amenable to surgery.The evidence is based on analysis of change in SEGA volume. Further clinical benefit, such as improvement in disease‑related symptoms, has not been demonstrated.
Intervention Type
Pharmaceutical
Solid/Hematological
Solid
Cancer Type
Brain, Central Nervous System
Therapy Types
Targeted Therapy
Regulatory
FDA
Regulatory Pathway: n/a
EMA
Regulatory Pathway: conditional approval
Availability in other countries
N/A
Focus Area
Cancer
ASMR
II
Medical Need
Unmet
SMR
IMPORTANT
Orphan Status
Yes