Novartis announced, that the US Food and Drug Administration (FDA) has approved Afinitor (everolimus) tablets for patients with subependymal giant cell astrocytoma (SEGA), a benign brain tumour associated with tuberous sclerosis (TS), who require therapeutic intervention but are not candidates for curative surgical resection.

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.

Prior to this FDA approval, the only treatment option for growing SEGAs, which primarily affect children and adolescents, was brain surgery. Tuberous sclerosis is a genetic disorder affecting approximately 25,000 to 40,000 people in the US that may cause benign tumours to form in vital organs. SEGAs, benign brain tumours, occur in up to 20 per cent of patients with TS.

"Today's FDA decision is an important milestone for the children and adults living with SEGA associated with tuberous sclerosis," said Hervé Hoppenot, president of Novartis Oncology. "We are committed to furthering research for patients with tuberous sclerosis and will continue to work towards addressing their unmet medical needs."

In this study, nearly one-third of patients (32%) experienced a reduction of 50% or greater in the size of their largest SEGA at six months relative to baseline. None of the patients developed a new SEGA while receiving Afinitor.

"SEGAs can be challenging for individuals with tuberous sclerosis and for the whole family, which is why we are encouraged to see ongoing research and new treatment options like Afinitor for these individuals," said Vicky Whittemore, vice president and chief scientific officer of the patient advocacy group the Tuberous Sclerosis Alliance.

For the treatment of patients with SEGA associated with TS, Afinitor received FDA priority review status, which is granted to drugs that offer major advances in treatment. This 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 is continuing to study the efficacy and clinical benefit of Afinitor for patients with SEGA associated with TS in a phase-III trial.
Novartis has submitted marketing applications for everolimus to the European Medicines Agency (EMA) and the Swiss Agency for Therapeutic Products (Swissmedic), and additional regulatory submissions are underway worldwide. If approved in the European Union (EU) for this indication, everolimus will be made available under the trade name Votubia.