Study at ASCO shows Novartis drug Afinitor first to shrink SEGA brain tumors in children and adults with tuberous sclerosis
Phase II study shows meaningful reduction in brain tumor size in 75% of patients with subependymal giant cell astrocytomas (SEGAs) from baseline to six months
Novartis announced that results from a Phase II study show Afinitor® (everolimus) tablets is the first medication in a clinical trial to decrease the size of subependymal giant cell astrocytomas (SEGAs), benign brain tumors associated with tuberous sclerosis (TS). In this study of 28 patients presented today at the 46th American Society of Clinical Oncology (ASCO) annual meeting in Chicago, 75% of patients experienced a reduction of 30% or greater in the size of their brain tumors from baseline to six months (p<0.001)[1].
Everolimus is approved under the trade name Afinitor® (everolimus) tablets for the treatment of patients with advanced renal cell carcinoma whose disease has progressed on or after treatment with vascular endothelial growth factor (VEGF)-targeted therapy.
SEGAs, occurring in 5-20% of patients with TS, primarily affect children and adolescents and can cause severe swelling in the brain or hydrocephalus. Currently, the only treatment option for patients with growing SEGAs is brain surgery. Patients enrolled in the study had evidence of established SEGA growth. The data show everolimus significantly decreased the size of SEGAs and no patient required surgery or developed new SEGAs while receiving everolimus.
These data formed the basis for a regulatory submission to the US Food and Drug Administration (FDA) for the treatment of patients with SEGA associated with TS. Everolimus has orphan drug designation for TS in the US. Orphan drugs are those developed to treat diseases affecting fewer than 200,000 people nationally[4],[5].
"Our hope is to offer these patients the first approved medication to treat SEGAs associated with tuberous sclerosis," said Herve Hoppenot, President of Novartis Oncology. "As part of our worldwide everolimus development program, Novartis has launched the EXIST Phase III trial program to continue to evaluate the impact of everolimus in the fight against tuberous sclerosis."
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