Novartis Pharmaceuticals Corporation ("Novartis") announced today 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).
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.
Tuberous sclerosis is a genetic disorder that causes tumors to form in many vital organs. It is estimated to affect 25,000 to 40,000 people in the US and one to two million worldwide. These tumors can grow in any organ, but typically occur in the brain, kidneys, heart, eyes, lungs and skin. Common symptoms include seizures, mental retardation, autism, behavior problems and kidney and skin abnormalities.
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.