This accelerated approval of Afinitor is based on an open-label, single-arm, 28-patient study conducted by Cincinnati Children’s Hospital Medical Center.
The effectiveness of Afinitor is based on an analysis of change in SEGA volume. A Phase III study is underway that compares Afinitor to placebo to explore the clinical benefits of Afinitor for the treatment of patients with SEGA associated with TS.
The treatment of patients with SEGA indication was approved under the FDA’s accelerated approval program, which provides patients access to a treatment where previously there was an unmet medical need even though clinical benefit has yet to be confirmed.
Novartis Oncology president Herve Hoppenot said that FDA decision is an important milestone for the children and adults living with SEGA associated with tuberous sclerosis and they are committed to furthering research for patients with tuberous sclerosis and will continue to work towards addressing their unmet medical needs.
Afinitor is approved in the European Union for the treatment of patients with advanced renal cell carcinoma (RCC) whose disease has progressed on or after treatment with vascular endothelial growth factor (VEGF)-targeted therapy.
It also approved in the US for the treatment of patients with advanced RCC after failure of treatment with sunitinib or sorafenib.